A Phase 1 interventional study of Emicizumab in Healthy Volunteers and Hemophilia A, sponsored by Hoffmann-La Roche. Completed at 1 site in China. Open to male participants aged 20 Years to 45 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2019-09-30.
Sponsored by Hoffmann-La Roche · Phase 1, Interventional, and Treatment
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Among 22 studies testing emicizumab in Hemophilia A — 6 open to participants now, 8 under way, 3 of them phase 3. emicizumab · Hemophilia A.
Among the 22 studies testing emicizumab in Hemophilia A, this one is the 15th largest by enrollment, is one of 3 that started in 2018, is one of 2 in phase 1, and was the 8th registered.
844 studies on the registry are indexed under Hemophilia A; 136 are open to participants now.
Browse Hemophilia A studies →6 of the 22 studies testing emicizumab in Hemophilia A are open now, in 4 countries. Recruiting first, the newest first:
Sanofi · Sites in United States, Taiwan
Ages 12+ · Men only
Chulalongkorn University · Sites in Thailand
Ages 3–30 · Men only · Healthy volunteers
Bleeding and Clotting Disorders Institute Peoria, Illinois · Sites in United States
Ages 0–90
Indiana Hemophilia &Thrombosis Center, Inc. · Sites in United States
Ages 5–45 · Men only
Versiti Blood Health · Sites in United States
Ages 0–17
By country, each count listing its studies:
emicizumab: first FDA approval 2017 (HEMLIBRA), first EMA approval 2018. What it is approved for, and how studies give it, is on its page →
Of the 87 Hemophilia A studies recruiting now, 51 (59%) set no upper age limit and 31 (36%) stop at 75 or younger; 55 take children under 18; 10 take healthy volunteers.
This study: 20 Years to 45 Years · male · healthy volunteers too.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
22 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 2 · Phase 3 13 · Phase 4 6
First in 2015; this study’s year is marked. 3 started in 2018.
This study's enrollment of 16 is below the median of 30 across 499 interventional studies indexed under Hemophilia A.
Median enrollment 27 across the 22 interventional studies. Of the 12 that completed or were terminated, 11 (92%) have results posted.
This tab as a report to print or save as PDF → With an account.
12 of 22 have an industry lead sponsor. The 10 most frequent:
Hoffmann-La Roche is the lead sponsor of 2,101 studies on the registry; 88 are open to participants now.
Of its 330 completed or terminated interventional studies of FDA-regulated products, 248 (75%) have results posted.
25 studies in Hemophilia A by Hoffmann-La Roche: 5 open now, 8 under way, 4 of them phase 3. Of 15 finished, 12 (80%) have results posted.
Among the 25 studies in Hemophilia A by Hoffmann-La Roche, this one is the 15th largest by enrollment of the 20 interventional ones that give a count, is one of 4 that started in 2018, is one of 2 in phase 1, and was the 12th registered.
Hoffmann-La Roche’s Hemophilia A studies by the year they started, since 2012.
2 of the studies testing emicizumab in Hemophilia A that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05181618Hoffmann-La RocheA Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis | 2026-11-19 |
| NCT05500807Bleeding and Clotting Disorders Institute Peoria, IllinoisEmicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A | 2027-06 |
Add all 2 to your calendar (.ics) → With an account.
Among the 22 studies testing emicizumab in Hemophilia A, from their registry updates:
Among Hoffmann-La Roche’s 25 in Hemophilia A:
This tab as a report to print or save as PDF → With an account.
This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Emicizumab
Participants will receive a single 1 milligram per kilogram of body weight (mg/kg) subcutaneous dose of emicizumab on Day 1.
Also known as: RO5534262
Maximum Observed Plasma Concentration (Cmax) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Time to Cmax (Tmax) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Apparent Terminal Half-Life (t1/2) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Apparent Clearance (CL/F) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Apparent Volume of Distribution (Vz/F) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Mean Residence Time (MRT) of Emicizumab
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
Time frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade
The WHO Toxicity Grading Scale was used for assessing adverse event severity. Any adverse event not specifically listed in the WHO Toxicity Grading Scale was assessed according to the following levels of severity: Grade 1 is mild; Grade 2 is moderate; Grade 3 is severe; and Grade 4 is life-threatening. Investigator text for adverse events were encoded using the Medical Dictionary for Regulatory Activities (MedDRA) version 21.1. After informed consent had been obtained but prior to initiation of study drug, only serious adverse events (SAEs) caused by a protocol-mandated intervention were to have been reported. After initiation of study drug, all adverse events, regardless of relationship to study drug, were to have been reported until the participant completed his last study visit. After this period, any SAEs believed to be related to prior study drug treatment were to be reported.
Time frame: From screening to study completion (20 weeks)
Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study
Participants were considered to be 'ADA Negative (Treatment Unaffected)' if baseline and all post-baseline samples were negative, or if they were ADA positive at baseline but did not have any post-baseline samples with a titer that was at least 4-fold greater than the titer of the baseline sample. 'Total ADA Positive' is the sum of all participants who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Induced)', those who were ADA negative at baseline and tested positive for ADA following study drug administration; and 'ADA Positive (Treatment Boosted)', those who were pre-dose ADA positive and had post-baseline samples with a titer that was at least 4-fold greater compared to the baseline measurement.
Time frame: Predose at Baseline (Day 1) and postdose on Days 57 and 113
Number of Participants With Laboratory Test Abnormalities
The number of participants with a laboratory abnormality during treatment (numerator) is reported among the 'number analyzed' in the table below (denominator), which represents the number of participants without that abnormality at baseline (last observation prior to initiation of study drug). Note that samples from all participants were analyzed for each laboratory parameter. Values falling above or below the Roche predefined standard reference range were laboratory abnormalities labelled accordingly as 'high' or 'low'. Not every laboratory abnormality qualified as an adverse event; only if it was accompanied by clinical symptoms, resulted in a change in study treatment or in a medical intervention, or was clinically significant in the investigator's judgment. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase
Time frame: Baseline and Days 2, 4, 8, 11, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57, and 113
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57 and 113
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57 and 113
Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR)
The INR is a standardized measure of the prothrombin time. Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 11, 29, 57 and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin
Erythrocyte mean corpuscular hemoglobin (MCH) is a measure of the average amount of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume
Erythrocyte mean corpuscular volume is a measure of the average volume of a red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration
Erythrocyte mean corpuscular hemoglobin concentration (MCHC) is a measure of the average concentration of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1)
Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113
Number of Participants by Test Results for Blood in Urine by Timepoint
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for blood in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 8, 29, 57, 85, and 113
Number of Participants by Test Results for Glucose in Urine by Timepoint
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for glucose in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 8, 29, 57, 85, and 113
Number of Participants by Test Results for Protein in Urine by Timepoint
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for protein in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
Time frame: Baseline and Days 2, 8, 29, 57, 85, and 113
Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Change From Baseline in Vital Signs by Timepoint: Pulse Rate
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Change From Baseline in Vital Signs by Timepoint: Respiratory Rate
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary)
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113
Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in ECG Results by Timepoint: PR Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in ECG Results by Timepoint: QRS Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in ECG Results by Timepoint: QT Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula)
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula)
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Change From Baseline in ECG Results by Timepoint: RR Duration
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
Time frame: Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113
Number of Participants With Concomitant Medications
The original terms recorded by the investigator for concomitant medications were standardized by the sponsor by assigning preferred terms. The duration of treatment with the concomitant medications ranged from 1 day to 5 days. Except for 1 participant who was treated during the in-clinic period (Days -1 to 4), all other concomitant medications were recorded during the ambulatory period (Days 6 to 113).
Time frame: From screening to study completion (20 weeks)
| Milestone | Emicizumab |
|---|---|
| Started | 16 |
| Completed | 16 |
| Not completed | 0 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| microgram per milliliter (μg/mL) | Emicizumab |
|---|---|
| Maximum Observed Plasma Concentration (Cmax) of Emicizumab | 7.11 ± 1.77 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| μg*day/mL | Emicizumab |
|---|---|
| Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab | 287 ± 74.2 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| μg*day/mL | Emicizumab |
|---|---|
| AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab | 268 ± 63.7 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| day | Emicizumab |
|---|---|
| Time to Cmax (Tmax) of Emicizumab | 7.0 (3.0 to 15.0) |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| day | Emicizumab |
|---|---|
| Apparent Terminal Half-Life (t1/2) of Emicizumab | 26.7 ± 4.25 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| milliliter per day (mL/day) | Emicizumab |
|---|---|
| Apparent Clearance (CL/F) of Emicizumab | 235 ± 88 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| mL | Emicizumab |
|---|---|
| Apparent Volume of Distribution (Vz/F) of Emicizumab | 8870 ± 2950 |
Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.
| day | Emicizumab |
|---|---|
| Mean Residence Time (MRT) of Emicizumab | 40.26 ± 6.34 |
The WHO Toxicity Grading Scale was used for assessing adverse event severity. Any adverse event not specifically listed in the WHO Toxicity Grading Scale was assessed according to the following levels of severity: Grade 1 is mild; Grade 2 is moderate; Grade 3 is severe; and Grade 4 is life-threatening. Investigator text for adverse events were encoded using the Medical Dictionary for Regulatory Activities (MedDRA) version 21.1. After informed consent had been obtained but prior to initiation of study drug, only serious adverse events (SAEs) caused by a protocol-mandated intervention were to have been reported. After initiation of study drug, all adverse events, regardless of relationship to study drug, were to have been reported until the participant completed his last study visit. After this period, any SAEs believed to be related to prior study drug treatment were to be reported.
| Participants | Emicizumab |
|---|---|
| Any Adverse Event - Any Grade | 14 |
| Any Adverse Event - Grade 1 | 7 |
| Any Adverse Event - Grade 2 | 7 |
| Any Adverse Event - Grade 3 | 0 |
| Any Adverse Event - Grade 4 | 0 |
| Leukopenia - Grade 1 | 1 |
| Diarrhoea - Grade 1 | 1 |
| Flatulence - Grade 1 | 1 |
| Mouth ulceration - Grade 2 | 1 |
| Toothache - Grade 1 | 1 |
| Conjunctivitis - Grade 2 | 1 |
| Gastroenteritis - Grade 2 | 1 |
| Pericoronitis - Grade 1 | 1 |
| Upper respiratory tract infection - Grade 1 | 3 |
| Upper respiratory tract infection - Grade 2 | 1 |
| Injury - Grade 2 | 1 |
| Road traffic accident - Grade 2 | 1 |
| Blood bilirubin increased - Grade 1 | 1 |
| Blood creatinine phosphokinase increased - Grade 1 | 4 |
| Blood triglycerides increased - Grade 1 | 1 |
| Blood uric acid increased - Grade 1 | 1 |
| C-reactive protein increased - Grade 1 | 2 |
| White blood cell count decreased - Grade 1 | 1 |
| White blood cell count increased - Grade 1 | 1 |
| Hypertriglyceridaemia - Grade 1 | 1 |
| Hypokalaemia - Grade 2 | 1 |
| Cough - Grade 1 | 1 |
| Dry throat - Grade 1 | 1 |
| Nasal congestion - Grade 1 | 1 |
| Nasal obstruction - Grade 2 | 1 |
| Oropharyngeal pain - Grade 1 | 3 |
| Rash - Grade 1 | 1 |
Participants were considered to be 'ADA Negative (Treatment Unaffected)' if baseline and all post-baseline samples were negative, or if they were ADA positive at baseline but did not have any post-baseline samples with a titer that was at least 4-fold greater than the titer of the baseline sample. 'Total ADA Positive' is the sum of all participants who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Induced)', those who were ADA negative at baseline and tested positive for ADA following study drug administration; and 'ADA Positive (Treatment Boosted)', those who were pre-dose ADA positive and had post-baseline samples with a titer that was at least 4-fold greater compared to the baseline measurement.
| Participants | Emicizumab |
|---|---|
| Baseline - ADA Positive | 0 |
| Baseline - ADA Negative | 16 |
| Day 57 - ADA Positive | 0 |
| Day 57 - ADA Negative | 16 |
| Day 113 - ADA Positive | 1 |
| Day 113 - ADA Negative | 15 |
| Overall - ADA Negative (Treatment Unaffected) | 15 |
| Overall - Total ADA Positive (Induced + Boosted) | 1 |
| Overall - ADA Positive (Treatment Induced) | 1 |
| Overall - ADA Positive (Treatment Boosted) | 0 |
The number of participants with a laboratory abnormality during treatment (numerator) is reported among the 'number analyzed' in the table below (denominator), which represents the number of participants without that abnormality at baseline (last observation prior to initiation of study drug). Note that samples from all participants were analyzed for each laboratory parameter. Values falling above or below the Roche predefined standard reference range were laboratory abnormalities labelled accordingly as 'high' or 'low'. Not every laboratory abnormality qualified as an adverse event; only if it was accompanied by clinical symptoms, resulted in a change in study treatment or in a medical intervention, or was clinically significant in the investigator's judgment. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase
| Participants | Emicizumab |
|---|---|
| Albumin - Low | 1 |
| Alkaline Phosphatase - High | 2 |
| SGPT/ALT - Low | 2 |
| SGOT/AST - Low | 5 |
| Blood Urea Nitrogen - Low | 1 |
| Blood Urea Nitrogen - High | 1 |
| Chloride - Low | 2 |
| Cholesterol - Low | 5 |
| Cholesterol - High | 1 |
| Creatine Kinase - High | 3 |
| C-Reactive Protein - High | 6 |
| Lactate Dehydrogenase - Low | 2 |
| Potassium - Low | 1 |
| Sodium - Low | 6 |
| Bilirubin - High | 2 |
| Protein, Total - Low | 3 |
| Triglycerides (Fasting) - High | 6 |
| Uric Acid - Low | 2 |
| Uric Acid - High | 4 |
| Fibrinogen - Low | 2 |
| Activated Partial Thromboplastin Time - Low | 13 |
| Basophils, Absolute Count - High | 4 |
| Eosinophils, Absolute Count - Low | 1 |
| Eosinophils, Absolute Count - High | 2 |
| Hematocrit - Low | 3 |
| Hemoglobin - High | 1 |
| Lymphocytes, Absolute Count - Low | 4 |
| Lymphocytes, Absolute Count - High | 1 |
| Erythrocyte Mean Corpuscular Hemoglobin (HGB)-High | 1 |
| Erythro. Mean Corpuscular HGB Concentration - High | 8 |
| Monocytes, Absolute Count - High | 2 |
| Neutrophils, Total, Absolute Count - Low | 2 |
| Neutrophils, Total, Absolute Count - High | 2 |
| Platelet - High | 1 |
| Red Blood Cell Count - Low | 3 |
| White Blood Cell Count - Low | 2 |
| Urine pH - High | 9 |
| Urine Specific Gravity - Low | 6 |
| Urine Specific Gravity - High | 9 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| gram per Liter (g/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 45.43 ± 1.834 |
| Change From BL at Day 2 | -0.43 ± 2.391 |
| Change From BL at Day 4 | 1.03 ± 2.016 |
| Change From BL at Day 8 | 0.29 ± 2.095 |
| Change From BL at Day 15 | 0.08 ± 1.928 |
| Change From BL at Day 29 | -0.18 ± 1.602 |
| Change From BL at Day 43 | -0.41 ± 1.242 |
| Change From BL at Day 57 | -1.24 ± 2.298 |
| Change From BL at Day 71 | -0.58 ± 1.983 |
| Change From BL at Day 85 | -1.56 ± 2.308 |
| Change From BL at Day 113 | -0.26 ± 1.732 |
| Post-BL Minimum Change From BL | -2.87 ± 1.967 |
| Post-BL Maximum Change From BL | 1.98 ± 1.521 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| unit per Liter (U/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 85.38 ± 19.500 |
| Change From BL at Day 2 | 0.50 ± 6.792 |
| Change From BL at Day 4 | 2.50 ± 6.542 |
| Change From BL at Day 8 | 1.06 ± 5.813 |
| Change From BL at Day 15 | 2.38 ± 9.150 |
| Change From BL at Day 29 | 1.44 ± 8.959 |
| Change From BL at Day 43 | 0.56 ± 8.756 |
| Change From BL at Day 57 | -3.69 ± 9.918 |
| Change From BL at Day 71 | -0.94 ± 11.198 |
| Change From BL at Day 85 | -4.50 ± 7.589 |
| Change From BL at Day 113 | -0.38 ± 11.684 |
| Post-BL Minimum Change From BL | -9.81 ± 7.556 |
| Post-BL Maximum Change From BL | 9.94 ± 7.398 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| micromole per Liter (μmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 13.83 ± 7.556 |
| Change From BL at Day 2 | 0.82 ± 2.921 |
| Change From BL at Day 4 | -0.42 ± 4.470 |
| Change From BL at Day 8 | -1.10 ± 3.713 |
| Change From BL at Day 15 | -0.61 ± 5.909 |
| Change From BL at Day 29 | -0.56 ± 4.688 |
| Change From BL at Day 43 | -0.25 ± 3.709 |
| Change From BL at Day 57 | -0.96 ± 4.034 |
| Change From BL at Day 71 | 1.67 ± 7.467 |
| Change From BL at Day 85 | 0.54 ± 3.861 |
| Change From BL at Day 113 | -0.12 ± 5.570 |
| Post-BL Minimum Change From BL | -3.94 ± 4.749 |
| Post-BL Maximum Change From BL | 5.54 ± 5.909 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 5.03 ± 0.425 |
| Change From BL at Day 2 | -0.33 ± 0.384 |
| Change From BL at Day 4 | -0.22 ± 0.500 |
| Change From BL at Day 8 | -0.16 ± 0.441 |
| Change From BL at Day 15 | -0.27 ± 0.382 |
| Change From BL at Day 29 | -0.08 ± 0.364 |
| Change From BL at Day 43 | -0.08 ± 0.435 |
| Change From BL at Day 57 | -0.22 ± 0.515 |
| Change From BL at Day 71 | -0.20 ± 0.322 |
| Change From BL at Day 85 | -0.25 ± 0.346 |
| Change From BL at Day 113 | -0.21 ± 0.470 |
| Post-BL Minimum Change From BL | -0.64 ± 0.388 |
| Post-BL Maximum Change From BL | 0.24 ± 0.386 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 4.46 ± 0.848 |
| Change From BL at Day 2 | -0.58 ± 0.877 |
| Change From BL at Day 4 | -0.73 ± 0.649 |
| Change From BL at Day 8 | 0.26 ± 1.074 |
| Change From BL at Day 15 | 0.43 ± 1.543 |
| Change From BL at Day 29 | 0.43 ± 1.071 |
| Change From BL at Day 43 | 0.38 ± 0.959 |
| Change From BL at Day 57 | 0.39 ± 0.874 |
| Change From BL at Day 71 | 0.36 ± 0.958 |
| Change From BL at Day 85 | 0.16 ± 0.888 |
| Change From BL at Day 113 | 0.13 ± 0.573 |
| Post-BL Minimum Change From BL | -1.07 ± 0.587 |
| Post-BL Maximum Change From BL | 1.42 ± 0.970 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| milligram per Liter (mg/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 0.50 ± 0.568 |
| Change From BL at Day 2 | 0.23 ± 0.456 |
| Change From BL at Day 4 | 0.11 ± 0.477 |
| Change From BL at Day 8 | 0.54 ± 1.721 |
| Change From BL at Day 15 | 0.75 ± 1.197 |
| Change From BL at Day 29 | 1.01 ± 1.289 |
| Change From BL at Day 43 | 0.60 ± 1.849 |
| Change From BL at Day 57 | 0.27 ± 0.827 |
| Change From BL at Day 71 | 0.13 ± 0.410 |
| Change From BL at Day 85 | 0.12 ± 0.595 |
| Change From BL at Day 113 | 1.24 ± 3.517 |
| Post-BL Minimum Change From BL | -0.20 ± 0.397 |
| Post-BL Maximum Change From BL | 3.14 ± 3.641 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 104.69 ± 1.957 |
| Change From BL at Day 2 | -0.69 ± 2.549 |
| Change From BL at Day 4 | -0.63 ± 2.363 |
| Change From BL at Day 8 | 0.13 ± 2.579 |
| Change From BL at Day 15 | 0.06 ± 2.175 |
| Change From BL at Day 29 | -1.56 ± 2.279 |
| Change From BL at Day 43 | -0.06 ± 1.731 |
| Change From BL at Day 57 | 0.75 ± 2.769 |
| Change From BL at Day 71 | -0.50 ± 2.757 |
| Change From BL at Day 85 | 0.13 ± 2.630 |
| Change From BL at Day 113 | -0.13 ± 2.217 |
| Post-BL Minimum Change From BL | -2.94 ± 2.016 |
| Post-BL Maximum Change From BL | 2.31 ± 2.120 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 3.88 ± 1.103 |
| Change From BL at Day 2 | 0.02 ± 0.393 |
| Change From BL at Day 4 | 0.07 ± 0.439 |
| Change From BL at Day 8 | -0.03 ± 0.383 |
| Change From BL at Day 15 | -0.04 ± 0.451 |
| Change From BL at Day 29 | 0.03 ± 0.372 |
| Change From BL at Day 43 | -0.06 ± 0.441 |
| Change From BL at Day 57 | -0.04 ± 0.459 |
| Change From BL at Day 71 | -0.01 ± 0.466 |
| Change From BL at Day 85 | -0.13 ± 0.515 |
| Change From BL at Day 113 | 0.01 ± 0.394 |
| Post-BL Minimum Change From BL | -0.47 ± 0.468 |
| Post-BL Maximum Change From BL | 0.47 ± 0.222 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| unit per Liter (U/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 121.63 ± 81.915 |
| Change From BL at Day 2 | -41.13 ± 54.760 |
| Change From BL at Day 4 | -25.81 ± 107.494 |
| Change From BL at Day 8 | -22.19 ± 54.034 |
| Change From BL at Day 15 | -0.94 ± 39.834 |
| Change From BL at Day 29 | 8.50 ± 122.645 |
| Change From BL at Day 43 | -21.00 ± 53.551 |
| Change From BL at Day 57 | 8.00 ± 152.217 |
| Change From BL at Day 71 | -19.63 ± 51.798 |
| Change From BL at Day 85 | -20.00 ± 77.283 |
| Change From BL at Day 113 | -18.81 ± 71.191 |
| Post-BL Minimum Change From BL | -52.38 ± 72.902 |
| Post-BL Maximum Change From BL | 92.50 ± 155.002 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| micromole per Liter (μmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 83.19 ± 7.195 |
| Change From BL at Day 2 | -0.56 ± 7.071 |
| Change From BL at Day 4 | -0.31 ± 5.147 |
| Change From BL at Day 8 | -1.25 ± 4.919 |
| Change From BL at Day 15 | -2.00 ± 9.452 |
| Change From BL at Day 29 | -3.69 ± 4.672 |
| Change From BL at Day 43 | -2.06 ± 5.434 |
| Change From BL at Day 57 | -2.38 ± 5.488 |
| Change From BL at Day 71 | -1.06 ± 7.344 |
| Change From BL at Day 85 | -1.50 ± 4.662 |
| Change From BL at Day 113 | -0.19 ± 6.327 |
| Post-BL Minimum Change From BL | -9.25 ± 5.170 |
| Post-BL Maximum Change From BL | 6.13 ± 6.109 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| micromole per Liter (μmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 1.63 ± 0.567 |
| Change From BL at Day 2 | 0.56 ± 0.395 |
| Change From BL at Day 4 | 0.29 ± 0.452 |
| Change From BL at Day 8 | 0.00 ± 0.327 |
| Change From BL at Day 15 | -0.07 ± 0.521 |
| Change From BL at Day 29 | 0.12 ± 0.548 |
| Change From BL at Day 43 | 0.12 ± 0.308 |
| Change From BL at Day 57 | -0.03 ± 0.342 |
| Change From BL at Day 71 | 0.23 ± 0.509 |
| Change From BL at Day 85 | 0.17 ± 0.515 |
| Change From BL at Day 113 | 0.07 ± 0.480 |
| Post-BL Minimum Change From BL | -0.39 ± 0.396 |
| Post-BL Maximum Change From BL | 0.78 ± 0.448 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| unit per Liter (U/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 19.94 ± 10.951 |
| Change From BL at Day 2 | -0.50 ± 1.862 |
| Change From BL at Day 4 | 0.13 ± 1.996 |
| Change From BL at Day 8 | 0.19 ± 3.016 |
| Change From BL at Day 15 | -0.13 ± 10.379 |
| Change From BL at Day 29 | -0.13 ± 3.202 |
| Change From BL at Day 43 | -0.13 ± 3.845 |
| Change From BL at Day 57 | -0.25 ± 4.712 |
| Change From BL at Day 71 | 0.13 ± 5.353 |
| Change From BL at Day 85 | -0.69 ± 3.790 |
| Change From BL at Day 113 | 1.75 ± 5.756 |
| Post-BL Minimum Change From BL | -4.81 ± 7.901 |
| Post-BL Maximum Change From BL | 5.94 ± 6.319 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| unit per Liter (U/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 153.75 ± 22.228 |
| Change From BL at Day 2 | -10.13 ± 13.495 |
| Change From BL at Day 4 | -0.19 ± 15.246 |
| Change From BL at Day 8 | 3.19 ± 11.053 |
| Change From BL at Day 15 | 3.19 ± 17.505 |
| Change From BL at Day 29 | 6.56 ± 17.397 |
| Change From BL at Day 43 | 2.38 ± 11.730 |
| Change From BL at Day 57 | 1.69 ± 11.247 |
| Change From BL at Day 71 | 0.19 ± 15.003 |
| Change From BL at Day 85 | 2.63 ± 16.536 |
| Change From BL at Day 113 | -0.38 ± 16.141 |
| Post-BL Minimum Change From BL | -16.06 ± 11.168 |
| Post-BL Maximum Change From BL | 22.00 ± 13.765 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 4.27 ± 0.351 |
| Change From BL at Day 2 | 0.11 ± 0.401 |
| Change From BL at Day 4 | -0.03 ± 0.307 |
| Change From BL at Day 8 | 0.12 ± 0.316 |
| Change From BL at Day 15 | -0.02 ± 0.351 |
| Change From BL at Day 29 | -0.05 ± 0.337 |
| Change From BL at Day 43 | 0.06 ± 0.366 |
| Change From BL at Day 57 | 0.05 ± 0.507 |
| Change From BL at Day 71 | -0.04 ± 0.320 |
| Change From BL at Day 85 | -0.19 ± 0.317 |
| Change From BL at Day 113 | -0.05 ± 0.371 |
| Post-BL Minimum Change From BL | -0.47 ± 0.311 |
| Post-BL Maximum Change From BL | 0.45 ± 0.248 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| gram per Liter (g/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 70.55 ± 3.485 |
| Change From BL at Day 2 | -0.18 ± 4.502 |
| Change From BL at Day 4 | 2.69 ± 3.711 |
| Change From BL at Day 8 | 1.36 ± 3.458 |
| Change From BL at Day 15 | 0.81 ± 3.837 |
| Change From BL at Day 29 | 1.03 ± 2.632 |
| Change From BL at Day 43 | -0.03 ± 2.452 |
| Change From BL at Day 57 | -1.41 ± 3.502 |
| Change From BL at Day 71 | 0.17 ± 3.720 |
| Change From BL at Day 85 | -1.13 ± 4.063 |
| Change From BL at Day 113 | 0.71 ± 3.779 |
| Post-BL Minimum Change From BL | -3.95 ± 3.488 |
| Post-BL Maximum Change From BL | 4.54 ± 2.451 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate transaminase
| unit per Liter (U/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 16.63 ± 2.918 |
| Change From BL at Day 2 | -0.81 ± 2.007 |
| Change From BL at Day 4 | 1.13 ± 2.941 |
| Change From BL at Day 8 | 0.69 ± 3.240 |
| Change From BL at Day 15 | 0.19 ± 3.728 |
| Change From BL at Day 29 | 1.13 ± 3.304 |
| Change From BL at Day 43 | -0.25 ± 1.770 |
| Change From BL at Day 57 | 0.69 ± 3.219 |
| Change From BL at Day 71 | -0.31 ± 2.798 |
| Change From BL at Day 85 | -0.63 ± 2.778 |
| Change From BL at Day 113 | -0.19 ± 3.920 |
| Post-BL Minimum Change From BL | -2.94 ± 2.594 |
| Post-BL Maximum Change From BL | 4.75 ± 3.416 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug. SGPT/ALT = serum glutamic-pyruvic transaminase/alanine transaminase
| unit per Liter (U/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 15.63 ± 6.449 |
| Change From BL at Day 2 | -0.31 ± 2.798 |
| Change From BL at Day 4 | 2.19 ± 5.419 |
| Change From BL at Day 8 | 2.19 ± 3.953 |
| Change From BL at Day 15 | -0.13 ± 7.702 |
| Change From BL at Day 29 | 0.25 ± 6.340 |
| Change From BL at Day 43 | -1.69 ± 5.199 |
| Change From BL at Day 57 | -1.13 ± 6.490 |
| Change From BL at Day 71 | -0.50 ± 6.408 |
| Change From BL at Day 85 | -1.13 ± 5.560 |
| Change From BL at Day 113 | -0.63 ± 7.571 |
| Post-BL Minimum Change From BL | -5.13 ± 6.206 |
| Post-BL Maximum Change From BL | 8.25 ± 6.943 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 140.56 ± 1.590 |
| Change From BL at Day 2 | -0.44 ± 1.931 |
| Change From BL at Day 4 | -0.44 ± 1.931 |
| Change From BL at Day 8 | -0.06 ± 2.594 |
| Change From BL at Day 15 | 0.25 ± 1.612 |
| Change From BL at Day 29 | -1.75 ± 2.671 |
| Change From BL at Day 43 | -0.13 ± 2.062 |
| Change From BL at Day 57 | 0.06 ± 2.932 |
| Change From BL at Day 71 | -1.25 ± 3.130 |
| Change From BL at Day 85 | -0.38 ± 2.705 |
| Change From BL at Day 113 | -0.13 ± 1.893 |
| Post-BL Minimum Change From BL | -3.38 ± 2.391 |
| Post-BL Maximum Change From BL | 2.31 ± 1.922 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| millimole per Liter (mmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 0.98 ± 0.446 |
| Change From BL at Day 2 | 0.37 ± 0.377 |
| Change From BL at Day 4 | 0.57 ± 0.517 |
| Change From BL at Day 8 | 0.03 ± 0.350 |
| Change From BL at Day 15 | 0.22 ± 0.497 |
| Change From BL at Day 29 | 0.15 ± 0.382 |
| Change From BL at Day 43 | 0.17 ± 0.278 |
| Change From BL at Day 57 | 0.27 ± 0.726 |
| Change From BL at Day 71 | 0.14 ± 0.314 |
| Change From BL at Day 85 | 0.28 ± 0.395 |
| Change From BL at Day 113 | 0.33 ± 0.429 |
| Post-BL Minimum Change From BL | -0.19 ± 0.318 |
| Post-BL Maximum Change From BL | 1.02 ± 0.490 |
Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| micromole per Liter (μmol/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 323.44 ± 52.081 |
| Change From BL at Day 2 | 21.38 ± 28.329 |
| Change From BL at Day 4 | -15.06 ± 38.979 |
| Change From BL at Day 8 | -2.88 ± 35.955 |
| Change From BL at Day 15 | 2.44 ± 49.536 |
| Change From BL at Day 29 | 2.31 ± 32.946 |
| Change From BL at Day 43 | 9.63 ± 45.045 |
| Change From BL at Day 57 | 9.38 ± 53.799 |
| Change From BL at Day 71 | 33.81 ± 84.440 |
| Change From BL at Day 85 | 19.25 ± 50.464 |
| Change From BL at Day 113 | 34.56 ± 62.406 |
| Post-BL Minimum Change From BL | -39.94 ± 25.870 |
| Post-BL Maximum Change From BL | 79.25 ± 62.143 |
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| second | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 31.63 ± 2.111 |
| Change From BL at Day 2 | -2.79 ± 1.626 |
| Change From BL at Day 11 | -4.82 ± 1.466 |
| Change From BL at Day 29 | -3.63 ± 1.643 |
| Change From BL at Day 57 | -2.14 ± 1.177 |
| Change From BL at Day 113 | -0.70 ± 1.517 |
| Post-BL Minimum Change From BL | -4.83 ± 1.482 |
| Post-BL Maximum Change From BL | -0.56 ± 1.463 |
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| gram per Liter (g/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 2.43 ± 0.410 |
| Change From BL at Day 2 | 0.18 ± 0.281 |
| Change From BL at Day 11 | 0.28 ± 0.314 |
| Change From BL at Day 29 | 0.11 ± 0.312 |
| Change From BL at Day 57 | 0.05 ± 0.285 |
| Change From BL at Day 113 | 0.07 ± 0.438 |
| Post-BL Minimum Change From BL | -0.17 ± 0.222 |
| Post-BL Maximum Change From BL | 0.53 ± 0.290 |
Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| second | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 11.33 ± 0.685 |
| Change From BL at Day 2 | 0.00 ± 0.502 |
| Change From BL at Day 11 | -0.09 ± 0.463 |
| Change From BL at Day 29 | -0.18 ± 0.455 |
| Change From BL at Day 57 | -0.18 ± 0.508 |
| Change From BL at Day 113 | -0.17 ± 0.632 |
| Post-BL Minimum Change From BL | -0.58 ± 0.457 |
| Post-BL Maximum Change From BL | 0.37 ± 0.480 |
The INR is a standardized measure of the prothrombin time. Blood samples were collected from participants at the indicated timepoints for evaluation of coagulation parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| INR of prothrombin time (sec/sec) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 1.06 ± 0.062 |
| Change From BL at Day 2 | 0.00 ± 0.044 |
| Change From BL at Day 11 | -0.01 ± 0.043 |
| Change From BL at Day 29 | -0.02 ± 0.042 |
| Change From BL at Day 57 | -0.02 ± 0.046 |
| Change From BL at Day 113 | -0.02 ± 0.057 |
| Post-BL Minimum Change From BL | -0.05 ± 0.042 |
| Post-BL Maximum Change From BL | 0.03 ± 0.043 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 0.02 ± 0.018 |
| Change From BL at Day 2 | 0.00 ± 0.013 |
| Change From BL at Day 4 | 0.01 ± 0.018 |
| Change From BL at Day 8 | 0.01 ± 0.029 |
| Change From BL at Day 15 | 0.01 ± 0.022 |
| Change From BL at Day 29 | 0.01 ± 0.014 |
| Change From BL at Day 43 | 0.01 ± 0.023 |
| Change From BL at Day 57 | 0.01 ± 0.016 |
| Change From BL at Day 71 | 0.01 ± 0.022 |
| Change From BL at Day 85 | 0.00 ± 0.021 |
| Change From BL at Day 113 | 0.01 ± 0.019 |
| Post-BL Minimum Change From BL | -0.01 ± 0.016 |
| Post-BL Maximum Change From BL | 0.03 ± 0.021 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 0.14 ± 0.122 |
| Change From BL at Day 2 | 0.02 ± 0.063 |
| Change From BL at Day 4 | 0.01 ± 0.066 |
| Change From BL at Day 8 | 0.00 ± 0.066 |
| Change From BL at Day 15 | -0.01 ± 0.074 |
| Change From BL at Day 29 | -0.01 ± 0.055 |
| Change From BL at Day 43 | 0.00 ± 0.101 |
| Change From BL at Day 57 | -0.02 ± 0.055 |
| Change From BL at Day 71 | 0.01 ± 0.102 |
| Change From BL at Day 85 | -0.02 ± 0.063 |
| Change From BL at Day 113 | 0.02 ± 0.121 |
| Post-BL Minimum Change From BL | -0.06 ± 0.080 |
| Post-BL Maximum Change From BL | 0.07 ± 0.085 |
Erythrocyte mean corpuscular hemoglobin (MCH) is a measure of the average amount of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| picogram per cell | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 30.84 ± 1.815 |
| Change From BL at Day 2 | 0.23 ± 0.467 |
| Change From BL at Day 4 | 0.09 ± 0.473 |
| Change From BL at Day 8 | 0.26 ± 0.594 |
| Change From BL at Day 15 | -0.12 ± 0.472 |
| Change From BL at Day 29 | 0.04 ± 0.507 |
| Change From BL at Day 43 | 0.48 ± 0.493 |
| Change From BL at Day 57 | 0.28 ± 0.567 |
| Change From BL at Day 71 | 0.40 ± 0.625 |
| Change From BL at Day 85 | 0.39 ± 0.738 |
| Change From BL at Day 113 | 0.51 ± 0.933 |
| Post-BL Minimum Change From BL | -0.40 ± 0.412 |
| Post-BL Maximum Change From BL | 1.24 ± 0.730 |
Erythrocyte mean corpuscular volume is a measure of the average volume of a red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| femtoliter (fL) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 89.79 ± 3.689 |
| Change From BL at Day 2 | -0.14 ± 1.172 |
| Change From BL at Day 4 | 0.01 ± 0.954 |
| Change From BL at Day 8 | 0.21 ± 1.138 |
| Change From BL at Day 15 | 0.88 ± 1.036 |
| Change From BL at Day 29 | -0.08 ± 0.849 |
| Change From BL at Day 43 | 1.41 ± 1.670 |
| Change From BL at Day 57 | 1.04 ± 1.488 |
| Change From BL at Day 71 | 0.85 ± 1.498 |
| Change From BL at Day 85 | 0.86 ± 1.656 |
| Change From BL at Day 113 | 0.62 ± 1.775 |
| Post-BL Minimum Change From BL | -1.17 ± 0.899 |
| Post-BL Maximum Change From BL | 2.34 ± 1.138 |
Erythrocyte mean corpuscular hemoglobin concentration (MCHC) is a measure of the average concentration of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| gram per Liter (g/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 343.25 ± 8.790 |
| Change From BL at Day 2 | 3.13 ± 6.438 |
| Change From BL at Day 4 | 1.00 ± 7.239 |
| Change From BL at Day 8 | 2.31 ± 5.606 |
| Change From BL at Day 15 | -4.56 ± 6.562 |
| Change From BL at Day 29 | 0.69 ± 6.019 |
| Change From BL at Day 43 | 0.13 ± 8.853 |
| Change From BL at Day 57 | -0.75 ± 8.798 |
| Change From BL at Day 71 | 1.25 ± 6.372 |
| Change From BL at Day 85 | 1.13 ± 8.277 |
| Change From BL at Day 113 | 3.25 ± 8.004 |
| Post-BL Minimum Change From BL | -9.00 ± 6.143 |
| Post-BL Maximum Change From BL | 10.56 ± 6.099 |
Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| ratio of RBCs in blood (L/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 0.44 ± 0.020 |
| Change From BL at Day 2 | 0.01 ± 0.032 |
| Change From BL at Day 4 | 0.01 ± 0.024 |
| Change From BL at Day 8 | 0.00 ± 0.023 |
| Change From BL at Day 15 | 0.00 ± 0.018 |
| Change From BL at Day 29 | -0.01 ± 0.019 |
| Change From BL at Day 43 | 0.00 ± 0.025 |
| Change From BL at Day 57 | -0.01 ± 0.027 |
| Change From BL at Day 71 | 0.00 ± 0.027 |
| Change From BL at Day 85 | 0.00 ± 0.029 |
| Change From BL at Day 113 | 0.01 ± 0.029 |
| Post-BL Minimum Change From BL | -0.02 ± 0.020 |
| Post-BL Maximum Change From BL | 0.02 ± 0.022 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| gram per Liter (g/L) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 149.38 ± 8.065 |
| Change From BL at Day 2 | 3.94 ± 10.056 |
| Change From BL at Day 4 | 5.38 ± 7.571 |
| Change From BL at Day 8 | 0.88 ± 6.551 |
| Change From BL at Day 15 | -2.50 ± 6.439 |
| Change From BL at Day 29 | -2.50 ± 7.062 |
| Change From BL at Day 43 | -0.56 ± 6.345 |
| Change From BL at Day 57 | -1.94 ± 8.330 |
| Change From BL at Day 71 | 0.94 ± 7.505 |
| Change From BL at Day 85 | 0.88 ± 7.881 |
| Change From BL at Day 113 | 3.19 ± 9.086 |
| Post-BL Minimum Change From BL | -8.13 ± 6.076 |
| Post-BL Maximum Change From BL | 9.63 ± 5.608 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 1.65 ± 0.691 |
| Change From BL at Day 2 | 0.10 ± 0.381 |
| Change From BL at Day 4 | 0.37 ± 0.780 |
| Change From BL at Day 8 | 0.04 ± 0.595 |
| Change From BL at Day 15 | -0.09 ± 0.599 |
| Change From BL at Day 29 | -0.01 ± 0.422 |
| Change From BL at Day 43 | 0.07 ± 0.470 |
| Change From BL at Day 57 | 0.01 ± 0.464 |
| Change From BL at Day 71 | 0.06 ± 0.372 |
| Change From BL at Day 85 | 0.02 ± 0.397 |
| Change From BL at Day 113 | 0.04 ± 0.585 |
| Post-BL Minimum Change From BL | -0.35 ± 0.447 |
| Post-BL Maximum Change From BL | 0.61 ± 0.633 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 0.35 ± 0.087 |
| Change From BL at Day 2 | -0.04 ± 0.098 |
| Change From BL at Day 4 | -0.03 ± 0.111 |
| Change From BL at Day 8 | -0.04 ± 0.100 |
| Change From BL at Day 15 | -0.01 ± 0.136 |
| Change From BL at Day 29 | -0.01 ± 0.112 |
| Change From BL at Day 43 | -0.03 ± 0.082 |
| Change From BL at Day 57 | -0.04 ± 0.091 |
| Change From BL at Day 71 | -0.03 ± 0.075 |
| Change From BL at Day 85 | -0.04 ± 0.096 |
| Change From BL at Day 113 | -0.02 ± 0.112 |
| Post-BL Minimum Change From BL | -0.10 ± 0.080 |
| Post-BL Maximum Change From BL | 0.11 ± 0.112 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 3.57 ± 0.869 |
| Change From BL at Day 2 | -0.24 ± 0.959 |
| Change From BL at Day 4 | 0.12 ± 0.950 |
| Change From BL at Day 8 | -0.19 ± 0.809 |
| Change From BL at Day 15 | 0.19 ± 1.043 |
| Change From BL at Day 29 | 0.23 ± 1.063 |
| Change From BL at Day 43 | 0.06 ± 0.961 |
| Change From BL at Day 57 | -0.24 ± 0.758 |
| Change From BL at Day 71 | -0.06 ± 0.824 |
| Change From BL at Day 85 | -0.31 ± 0.686 |
| Change From BL at Day 113 | -0.27 ± 0.695 |
| Post-BL Minimum Change From BL | -0.86 ± 0.708 |
| Post-BL Maximum Change From BL | 1.16 ± 0.968 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 231.50 ± 47.880 |
| Change From BL at Day 2 | -2.13 ± 25.469 |
| Change From BL at Day 4 | -3.69 ± 22.330 |
| Change From BL at Day 8 | 0.06 ± 20.612 |
| Change From BL at Day 15 | 7.25 ± 23.023 |
| Change From BL at Day 29 | 12.50 ± 26.967 |
| Change From BL at Day 43 | 9.88 ± 30.265 |
| Change From BL at Day 57 | 10.94 ± 18.908 |
| Change From BL at Day 71 | 5.50 ± 34.131 |
| Change From BL at Day 85 | 0.50 ± 30.542 |
| Change From BL at Day 113 | 2.94 ± 23.778 |
| Post-BL Minimum Change From BL | -27.31 ± 17.984 |
| Post-BL Maximum Change From BL | 36.50 ± 26.473 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^12 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 4.86 ± 0.304 |
| Change From BL at Day 2 | 0.09 ± 0.327 |
| Change From BL at Day 4 | 0.16 ± 0.247 |
| Change From BL at Day 8 | -0.02 ± 0.235 |
| Change From BL at Day 15 | -0.07 ± 0.196 |
| Change From BL at Day 29 | -0.09 ± 0.209 |
| Change From BL at Day 43 | -0.09 ± 0.226 |
| Change From BL at Day 57 | -0.11 ± 0.289 |
| Change From BL at Day 71 | -0.04 ± 0.266 |
| Change From BL at Day 85 | -0.04 ± 0.334 |
| Change From BL at Day 113 | 0.02 ± 0.337 |
| Post-BL Minimum Change From BL | -0.31 ± 0.242 |
| Post-BL Maximum Change From BL | 0.26 ± 0.213 |
Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last sample prior to initiation of study drug.
| *10^9 cells per Liter | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 5.74 ± 1.565 |
| Change From BL at Day 2 | -0.17 ± 1.213 |
| Change From BL at Day 4 | 0.33 ± 1.285 |
| Change From BL at Day 8 | -0.20 ± 1.222 |
| Change From BL at Day 15 | 0.09 ± 1.400 |
| Change From BL at Day 29 | 0.21 ± 1.026 |
| Change From BL at Day 43 | 0.11 ± 1.162 |
| Change From BL at Day 57 | -0.27 ± 1.125 |
| Change From BL at Day 71 | -0.01 ± 1.006 |
| Change From BL at Day 85 | -0.34 ± 1.064 |
| Change From BL at Day 113 | -0.24 ± 1.332 |
| Post-BL Minimum Change From BL | -1.07 ± 1.071 |
| Post-BL Maximum Change From BL | 1.29 ± 0.997 |
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for blood in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
| Participants | Emicizumab |
|---|---|
| Baseline — 0 (Absent) | 15 |
| Baseline — +1 (Trace) | 1 |
| Baseline — +2 (Positive) | 0 |
| Baseline — +3/+4 (Strong Positive) | 0 |
| Day 2 — 0 (Absent) | 16 |
| Day 2 — +1 (Trace) | 0 |
| Day 2 — +2 (Positive) | 0 |
| Day 2 — +3/+4 (Strong Positive) | 0 |
| Day 8 — 0 (Absent) | 15 |
| Day 8 — +1 (Trace) | 1 |
| Day 8 — +2 (Positive) | 0 |
| Day 8 — +3/+4 (Strong Positive) | 0 |
| Day 29 — 0 (Absent) | 16 |
| Day 29 — +1 (Trace) | 0 |
| Day 29 — +2 (Positive) | 0 |
| Day 29 — +3/+4 (Strong Positive) | 0 |
| Day 57 — 0 (Absent) | 16 |
| Day 57 — +1 (Trace) | 0 |
| Day 57 — +2 (Positive) | 0 |
| Day 57 — +3/+4 (Strong Positive) | 0 |
| Day 85 — 0 (Absent) | 16 |
| Day 85 — +1 (Trace) | 0 |
| Day 85 — +2 (Positive) | 0 |
| Day 85 — +3/+4 (Strong Positive) | 0 |
| Day 113 — 0 (Absent) | 15 |
| Day 113 — +1 (Trace) | 1 |
| Day 113 — +2 (Positive) | 0 |
| Day 113 — +3/+4 (Strong Positive) | 0 |
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for glucose in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
| Participants | Emicizumab |
|---|---|
| Baseline — 0 (Absent) | 16 |
| Baseline — +1 (Trace) | 0 |
| Baseline — +2 (Positive) | 0 |
| Baseline — +3/+4 (Strong Positive) | 0 |
| Day 2 — 0 (Absent) | 16 |
| Day 2 — +1 (Trace) | 0 |
| Day 2 — +2 (Positive) | 0 |
| Day 2 — +3/+4 (Strong Positive) | 0 |
| Day 8 — 0 (Absent) | 16 |
| Day 8 — +1 (Trace) | 0 |
| Day 8 — +2 (Positive) | 0 |
| Day 8 — +3/+4 (Strong Positive) | 0 |
| Day 29 — 0 (Absent) | 16 |
| Day 29 — +1 (Trace) | 0 |
| Day 29 — +2 (Positive) | 0 |
| Day 29 — +3/+4 (Strong Positive) | 0 |
| Day 57 — 0 (Absent) | 16 |
| Day 57 — +1 (Trace) | 0 |
| Day 57 — +2 (Positive) | 0 |
| Day 57 — +3/+4 (Strong Positive) | 0 |
| Day 85 — 0 (Absent) | 16 |
| Day 85 — +1 (Trace) | 0 |
| Day 85 — +2 (Positive) | 0 |
| Day 85 — +3/+4 (Strong Positive) | 0 |
| Day 113 — 0 (Absent) | 15 |
| Day 113 — +1 (Trace) | 1 |
| Day 113 — +2 (Positive) | 0 |
| Day 113 — +3/+4 (Strong Positive) | 0 |
Urine samples were collected from participants at the indicated timepoints for evaluation of urinalysis parameters. The number of participants with test results for protein in urine of 0 (Absent), +1 (Trace), +2 (Positive), and +3/+4 (Strong Positive) at baseline and each timepoint are reported. Baseline was defined as the participant's last sample prior to initiation of study drug.
| Participants | Emicizumab |
|---|---|
| Baseline — 0 (Absent) | 16 |
| Baseline — +1 (Trace) | 0 |
| Baseline — +2 (Positive) | 0 |
| Baseline — +3/+4 (Strong Positive) | 0 |
| Day 2 — 0 (Absent) | 16 |
| Day 2 — +1 (Trace) | 0 |
| Day 2 — +2 (Positive) | 0 |
| Day 2 — +3/+4 (Strong Positive) | 0 |
| Day 8 — 0 (Absent) | 16 |
| Day 8 — +1 (Trace) | 0 |
| Day 8 — +2 (Positive) | 0 |
| Day 8 — +3/+4 (Strong Positive) | 0 |
| Day 29 — 0 (Absent) | 16 |
| Day 29 — +1 (Trace) | 0 |
| Day 29 — +2 (Positive) | 0 |
| Day 29 — +3/+4 (Strong Positive) | 0 |
| Day 57 — 0 (Absent) | 15 |
| Day 57 — +1 (Trace) | 1 |
| Day 57 — +2 (Positive) | 0 |
| Day 57 — +3/+4 (Strong Positive) | 0 |
| Day 85 — 0 (Absent) | 15 |
| Day 85 — +1 (Trace) | 1 |
| Day 85 — +2 (Positive) | 0 |
| Day 85 — +3/+4 (Strong Positive) | 0 |
| Day 113 — 0 (Absent) | 16 |
| Day 113 — +1 (Trace) | 0 |
| Day 113 — +2 (Positive) | 0 |
| Day 113 — +3/+4 (Strong Positive) | 0 |
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millimeters of mercury (mmHg) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 66.1 ± 5.77 |
| Change From BL at Day 2 | -0.8 ± 4.99 |
| Change From BL at Day 4 | 2.4 ± 5.21 |
| Change From BL at Day 6 | 3.2 ± 5.43 |
| Change From BL at Day 8 | 2.7 ± 5.41 |
| Change From BL at Day 11 | 1.4 ± 5.11 |
| Change From BL at Day 15 | 1.7 ± 4.24 |
| Change From BL at Day 22 | 3.2 ± 5.46 |
| Change From BL at Day 29 | 2.6 ± 5.67 |
| Change From BL at Day 36 | 1.3 ± 4.48 |
| Change From BL at Day 43 | 1.0 ± 5.18 |
| Change From BL at Day 50 | 3.4 ± 6.49 |
| Change From BL at Day 57 | -0.3 ± 4.64 |
| Change From BL at Day 71 | 1.1 ± 5.50 |
| Change From BL at Day 85 | 1.3 ± 4.52 |
| Change From BL at Day 113 | 1.3 ± 4.73 |
| Post-BL Minimum Change From BL | -5.2 ± 3.08 |
| Post-BL Maximum Change From BL | 8.1 ± 4.39 |
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| beats per minute | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 61.3 ± 7.25 |
| Change From BL at Day 2 | -0.6 ± 4.72 |
| Change From BL at Day 4 | 1.3 ± 4.90 |
| Change From BL at Day 6 | 5.8 ± 7.06 |
| Change From BL at Day 8 | 7.7 ± 8.58 |
| Change From BL at Day 11 | 9.9 ± 10.41 |
| Change From BL at Day 15 | 6.9 ± 5.71 |
| Change From BL at Day 22 | 10.2 ± 9.22 |
| Change From BL at Day 29 | 8.4 ± 9.24 |
| Change From BL at Day 36 | 6.3 ± 7.59 |
| Change From BL at Day 43 | 6.2 ± 7.93 |
| Change From BL at Day 50 | 10.4 ± 6.98 |
| Change From BL at Day 57 | 7.5 ± 6.75 |
| Change From BL at Day 71 | 4.7 ± 7.07 |
| Change From BL at Day 85 | 6.9 ± 9.48 |
| Change From BL at Day 113 | 7.4 ± 8.16 |
| Post-BL Minimum Change From BL | -3.1 ± 4.46 |
| Post-BL Maximum Change From BL | 18.1 ± 6.29 |
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| breaths per minute | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 15.5 ± 1.51 |
| Change From BL at Day 2 | 0.6 ± 1.50 |
| Change From BL at Day 4 | 0.3 ± 1.89 |
| Change From BL at Day 6 | -0.5 ± 2.00 |
| Change From BL at Day 8 | 0.8 ± 2.64 |
| Change From BL at Day 11 | 0.1 ± 2.55 |
| Change From BL at Day 15 | -0.1 ± 2.38 |
| Change From BL at Day 22 | -0.1 ± 2.60 |
| Change From BL at Day 29 | 0.2 ± 2.10 |
| Change From BL at Day 36 | 0.9 ± 1.54 |
| Change From BL at Day 43 | -0.3 ± 1.88 |
| Change From BL at Day 50 | 0.3 ± 2.32 |
| Change From BL at Day 57 | 0.2 ± 2.14 |
| Change From BL at Day 71 | -0.3 ± 2.02 |
| Change From BL at Day 85 | -0.4 ± 2.33 |
| Change From BL at Day 113 | 0.4 ± 2.47 |
| Post-BL Minimum Change From BL | -3.0 ± 1.83 |
| Post-BL Maximum Change From BL | 2.7 ± 1.82 |
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millimeters of mercury (mmHg) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 106.8 ± 7.26 |
| Change From BL at Day 2 | -1.6 ± 6.49 |
| Change From BL at Day 4 | 2.4 ± 5.82 |
| Change From BL at Day 6 | 3.6 ± 5.86 |
| Change From BL at Day 8 | 3.5 ± 8.16 |
| Change From BL at Day 11 | 5.1 ± 8.05 |
| Change From BL at Day 15 | 2.5 ± 4.97 |
| Change From BL at Day 22 | 4.8 ± 7.46 |
| Change From BL at Day 29 | 3.2 ± 8.84 |
| Change From BL at Day 36 | 2.2 ± 7.49 |
| Change From BL at Day 43 | 2.0 ± 5.97 |
| Change From BL at Day 50 | 5.2 ± 8.95 |
| Change From BL at Day 57 | 2.2 ± 7.19 |
| Change From BL at Day 71 | 1.2 ± 6.35 |
| Change From BL at Day 85 | 2.0 ± 8.68 |
| Change From BL at Day 113 | 1.1 ± 8.75 |
| Post-BL Minimum Change From BL | -4.6 ± 5.76 |
| Post-BL Maximum Change From BL | 10.9 ± 7.47 |
Vital signs were measured prior to blood sampling while the participant was in a supine position after they had been resting for at least 5 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| degrees Celsius (C) | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 36.20 ± 0.239 |
| Change From BL at Day 2 | -0.12 ± 0.281 |
| Change From BL at Day 4 | 0.01 ± 0.263 |
| Change From BL at Day 6 | -0.09 ± 0.532 |
| Change From BL at Day 8 | -0.08 ± 0.217 |
| Change From BL at Day 11 | -0.18 ± 0.414 |
| Change From BL at Day 15 | -0.06 ± 0.515 |
| Change From BL at Day 22 | -0.14 ± 0.352 |
| Change From BL at Day 29 | -0.11 ± 0.433 |
| Change From BL at Day 36 | -0.19 ± 0.313 |
| Change From BL at Day 43 | -0.16 ± 0.318 |
| Change From BL at Day 50 | -0.16 ± 0.418 |
| Change From BL at Day 57 | 0.02 ± 0.382 |
| Change From BL at Day 71 | -0.09 ± 0.360 |
| Change From BL at Day 85 | -0.21 ± 0.260 |
| Change From BL at Day 113 | -0.02 ± 0.297 |
| Post-BL Minimum Change From BL | -0.61 ± 0.260 |
| Post-BL Maximum Change From BL | 0.43 ± 0.357 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| beats per minute | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 59.90 ± 5.588 |
| Change From BL at Day 2 | -0.67 ± 3.656 |
| Change From BL at Day 4 | 2.50 ± 3.978 |
| Change From BL at Day 6 | 6.52 ± 6.491 |
| Change From BL at Day 8 | 8.63 ± 7.574 |
| Change From BL at Day 15 | 6.58 ± 4.250 |
| Change From BL at Day 29 | 8.10 ± 8.675 |
| Change From BL at Day 43 | 6.96 ± 7.946 |
| Change From BL at Day 57 | 6.42 ± 5.793 |
| Change From BL at Day 71 | 5.42 ± 6.976 |
| Change From BL at Day 85 | 8.25 ± 8.234 |
| Change From BL at Day 113 | 7.15 ± 6.442 |
| Post-BL Minimum Change From BL | -2.21 ± 2.638 |
| Post-BL Maximum Change From BL | 15.52 ± 6.259 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millisecond | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 160.21 ± 16.023 |
| Change From BL at Day 2 | 0.81 ± 12.767 |
| Change From BL at Day 4 | -0.10 ± 9.229 |
| Change From BL at Day 6 | -1.90 ± 9.998 |
| Change From BL at Day 8 | -4.13 ± 12.692 |
| Change From BL at Day 15 | -1.73 ± 14.577 |
| Change From BL at Day 29 | -4.10 ± 10.197 |
| Change From BL at Day 43 | -1.79 ± 13.850 |
| Change From BL at Day 57 | -1.52 ± 10.352 |
| Change From BL at Day 71 | -1.29 ± 9.008 |
| Change From BL at Day 85 | -5.02 ± 12.450 |
| Change From BL at Day 113 | -1.52 ± 6.576 |
| Post-BL Minimum Change From BL | -15.21 ± 11.517 |
| Post-BL Maximum Change From BL | 8.83 ± 9.000 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millisecond | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 98.15 ± 9.074 |
| Change From BL at Day 2 | 0.75 ± 5.514 |
| Change From BL at Day 4 | 1.40 ± 3.077 |
| Change From BL at Day 6 | -0.71 ± 3.052 |
| Change From BL at Day 8 | -0.48 ± 4.169 |
| Change From BL at Day 15 | -0.40 ± 3.221 |
| Change From BL at Day 29 | -1.00 ± 5.594 |
| Change From BL at Day 43 | -0.65 ± 2.327 |
| Change From BL at Day 57 | -1.54 ± 2.494 |
| Change From BL at Day 71 | 0.77 ± 3.429 |
| Change From BL at Day 85 | -0.46 ± 3.494 |
| Change From BL at Day 113 | -0.92 ± 3.782 |
| Post-BL Minimum Change From BL | -4.19 ± 3.592 |
| Post-BL Maximum Change From BL | 4.50 ± 3.475 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millisecond | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 398.31 ± 27.123 |
| Change From BL at Day 2 | -1.35 ± 11.351 |
| Change From BL at Day 4 | -10.00 ± 13.856 |
| Change From BL at Day 6 | -12.31 ± 19.791 |
| Change From BL at Day 8 | -15.69 ± 15.037 |
| Change From BL at Day 15 | -9.90 ± 13.857 |
| Change From BL at Day 29 | -11.83 ± 19.322 |
| Change From BL at Day 43 | -11.96 ± 13.863 |
| Change From BL at Day 57 | -7.56 ± 16.498 |
| Change From BL at Day 71 | -4.94 ± 19.933 |
| Change From BL at Day 85 | -9.75 ± 23.282 |
| Change From BL at Day 113 | -9.21 ± 20.349 |
| Post-BL Minimum Change From BL | -30.35 ± 14.706 |
| Post-BL Maximum Change From BL | 9.60 ± 12.064 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millisecond | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 398.04 ± 19.275 |
| Change From BL at Day 2 | -3.81 ± 10.367 |
| Change From BL at Day 4 | -2.29 ± 8.759 |
| Change From BL at Day 6 | 7.33 ± 8.549 |
| Change From BL at Day 8 | 10.08 ± 13.968 |
| Change From BL at Day 15 | 10.50 ± 10.870 |
| Change From BL at Day 29 | 12.69 ± 12.031 |
| Change From BL at Day 43 | 8.81 ± 13.699 |
| Change From BL at Day 57 | 12.00 ± 9.509 |
| Change From BL at Day 71 | 12.25 ± 12.194 |
| Change From BL at Day 85 | 15.27 ± 13.029 |
| Change From BL at Day 113 | 12.08 ± 13.229 |
| Post-BL Minimum Change From BL | -7.27 ± 8.817 |
| Post-BL Maximum Change From BL | 24.23 ± 11.165 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millisecond | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 397.90 ± 20.554 |
| Change From BL at Day 2 | -2.98 ± 8.968 |
| Change From BL at Day 4 | -4.94 ± 9.038 |
| Change From BL at Day 6 | 1.21 ± 9.739 |
| Change From BL at Day 8 | 1.31 ± 9.708 |
| Change From BL at Day 15 | 3.56 ± 10.109 |
| Change From BL at Day 29 | 4.33 ± 8.384 |
| Change From BL at Day 43 | 1.69 ± 8.274 |
| Change From BL at Day 57 | 5.31 ± 8.893 |
| Change From BL at Day 71 | 6.40 ± 11.109 |
| Change From BL at Day 85 | 6.71 ± 12.243 |
| Change From BL at Day 113 | 4.81 ± 12.931 |
| Post-BL Minimum Change From BL | -8.21 ± 8.174 |
| Post-BL Maximum Change From BL | 14.77 ± 10.366 |
The 12-lead electrocardiogram (ECG) measurements were collected in triplicate (three consecutive interpretable ECGs within 5 minutes) after the participant had been in a supine position for at least 10 minutes. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The post-baseline minimum and maximum change from baseline values are, respectively, the smallest and largest value changes obtained after baseline through the last study visit, including repeat and unscheduled tests. Baseline was defined as the participant's last value prior to initiation of study drug.
| millisecond | Emicizumab |
|---|---|
| Baseline (BL) - Value at Visit | 1001.06 ± 93.082 |
| Change From BL at Day 2 | 12.73 ± 66.160 |
| Change From BL at Day 4 | -36.04 ± 60.573 |
| Change From BL at Day 6 | -94.79 ± 99.788 |
| Change From BL at Day 8 | -119.67 ± 106.349 |
| Change From BL at Day 15 | -96.56 ± 67.805 |
| Change From BL at Day 29 | -114.08 ± 126.347 |
| Change From BL at Day 43 | -97.98 ± 109.011 |
| Change From BL at Day 57 | -92.98 ± 87.990 |
| Change From BL at Day 71 | -82.29 ± 112.001 |
| Change From BL at Day 85 | -115.87 ± 122.073 |
| Change From BL at Day 113 | -101.02 ± 98.269 |
| Post-BL Minimum Change From BL | -205.40 ± 91.026 |
| Post-BL Maximum Change From BL | 40.25 ± 49.296 |
The original terms recorded by the investigator for concomitant medications were standardized by the sponsor by assigning preferred terms. The duration of treatment with the concomitant medications ranged from 1 day to 5 days. Except for 1 participant who was treated during the in-clinic period (Days -1 to 4), all other concomitant medications were recorded during the ambulatory period (Days 6 to 113).
| Participants | Emicizumab |
|---|---|
| Total Participants with at Least 1 Treatment | 6 |
| Analgesics - Paracetamol | 1 |
| Antitrichomonal Agents - Tinidazole | 1 |
| Cephalosporin Antibiotics - Cefadroxil | 1 |
| Cephalosporin Antibiotics - Cefminox | 1 |
| Herbal, Homeopathic, and Dietary Supplements | 1 |
| Non-Steroidal Anti-Inflammatories - Pranoprofen | 1 |
| Proton Pump Inhibitors - Pantoprazole | 1 |
| Salicylates - Aspirin DL-Lysine | 1 |
| Supplements - Potassium Chloride | 1 |
| Supplements - Sodium Chloride | 1 |
| Vitamins and Minerals - Ascorbic Acid | 1 |
Collected over From screening to study completion (20 weeks). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Emicizumab | 0/16 (0%) | 0/16 (0%) | 14/16 (87.5%) |
| Event | Emicizumab |
|---|---|
| Upper respiratory tract infectionInfections and infestations | 4/16 |
| Blood creatinine phosphokinase increasedInvestigations | 4/16 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 3/16 |
| C-reactive protein increasedInvestigations | 2/16 |
| LeukopeniaBlood and lymphatic system disorders | 1/16 |
| DiarrhoeaGastrointestinal disorders | 1/16 |
| FlatulenceGastrointestinal disorders | 1/16 |
| Mouth ulcerationGastrointestinal disorders | 1/16 |
| ToothacheGastrointestinal disorders | 1/16 |
| ConjunctivitisInfections and infestations | 1/16 |
| Age, Continuous(years) | Emicizumab |
|---|---|
| Mean | 31.6 ± 6.7 |
| Sex: Female, Male(Participants) | Emicizumab |
|---|---|
| Female | 0 |
| Male | 16 |
| Ethnicity (NIH/OMB)(Participants) | Emicizumab |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 16 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Emicizumab |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 16 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 0 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Mar 2018 to primary completion in Sep 2018, 6 months.
Results were first posted in Sep 2019, 1 year after primary completion.
Of 37 industry-sponsored phase 1 interventional studies under Hemophilia A, half reached primary completion within 10 months of starting (the median), counting those still running; a quarter within 5 and three quarters within 18. This study's 6 months is below that.
Counting every sponsor, half of 46 studies reached it within 13 months; a quarter within 7 and three quarters within 26.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2019. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Emicizumab