A Phase 3 interventional study of PF-06741086 in Hemophilia A and Hemophilia B, sponsored by Pfizer. Completed at 62 sites in 20 countries. Open to male participants aged 12 Years to 74 Years. Per ClinicalTrials.gov, last updated 2026-08-14.
Sponsored by Pfizer · Phase 3, Interventional, and Treatment
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 188 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →Pfizer is the lead sponsor of 3,334 studies on the registry; 149 are open to participants now.
Of its 601 completed or terminated interventional studies of FDA-regulated products, 391 (65%) have results posted.
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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.
Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:
Exclusion Criteria
Regular, concomitant therapy with immunomodulatory drugs
- Ongoing or planned use of immune tolerance induction during the Observational Phase or Active Treatment Phase, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention during the Active Treatment Phase
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.
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.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
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.
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
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.
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.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
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.
CD4 count
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
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.
Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.
Drug: PF-06741086
300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria.
Model-Based Annualized Bleeding Rate (ABR) of Treated Bleeding Events: Inhibitor Cohort (Participants With Prior OD Therapy at OP)
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With Prior OD Therapy at OP)
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With RP at OP)
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Number of Participants With Adverse Events (AEs): Inhibitor and Non-Inhibitor Cohort
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious AEs and all other AEs.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Serious Adverse Events (SAEs): Inhibitor and Non-Inhibitor Cohort
AE: any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE: any untoward medical occurrence at any dose that resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Thrombotic Events: Inhibitor and Non-Inhibitor Cohort
Thrombotic events are when a blood clot (thrombus) forms in a blood vessel in the arm, leg, lung, or head and can be life-threatening. A thrombus can occur in veins (venous thrombosis) or arteries (arterial thrombosis).
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Thrombotic Microangiopathy: Inhibitor and Non-Inhibitor Cohort
Thrombotic microangiopathy: pathological state where micro-vessels are occluded by platelet rich thrombi leading to thrombocytopenia (low platelets) and microangiopathic haemolytic anaemia (red blood cell destruction) and potential end organ damage.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Disseminated Intravascular Coagulation/ Consumption Coagulopathy: Inhibitor and Non-Inhibitor Cohort
Disseminated intravascular coagulation is characterized by widespread clotting in small blood vessels, and consumption of platelets and clotting factors by the clots, leading to bleeding.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Anti-drug Antibodies (ADA) and Neutralizing Antibodies (NAb) Against PF- 06741086: Inhibitor and Non-Inhibitor Cohort
ADA positive: A participant with \>=1 treatment induced or treatment-boosted ADA response. NAb positive: An ADA positive participant with \>=1 treatment induced or treatment boosted NAb response.
Time frame: During prophylaxis treatment in ATP (12 months)
Number of Participants With Transient and Persistent ADA and NAb Against PF-06741086: Inhibitor and Non-Inhibitor Cohort
Persistent ADA: participant with treatment-induced or treatment-boosted ADA detected at 2 or more times during treatment including any follow-up, where first and last ADA positive samples separated by \>=16 weeks. Transient ADA: treatment-induced or treatment-boosted ADA detected at only 1 time during treatment or follow-up. Persistent NAb: NAb-positive participant with first and last positive NAb samples detected \>=16 weeks posttreatment, irrespective of any negative samples in between. Transient NAb: NAb-positive participant with (1) treatment-induced/treatment-boosted NAb sample detected at only 1 time posttreatment or (2) treatment-induced/treatment-boosted NAb samples detected at 2 or more times where first and last positive samples separated by \<16 weeks, and participant's last sample was NAb or ADA negative.
Time frame: During prophylaxis treatment in ATP (12 months)
Number of Participants With Injection Site Reactions (ISRs): Inhibitor and Non-Inhibitor Cohort
ISR included: injection site haematoma, injection site pain, injection site bruising, injection site erythema, injection site induration, injection site oedema, Injection site pruritus and Injection site swelling. ISR graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 where grade 1: tenderness with or without associated symptoms (warmth, erythema, itching), grade 2: pain, lipodystrophy, edema, phlebitis, grade 3: ulceration or necrosis, severe tissue damage, operative intervention indicated, grade 4: life-threatening consequences, urgent intervention indicated and grade 5: death. In this outcome measure only categories with non-zero values reported.
Time frame: During prophylaxis treatment in ATP (12 months)
Number of Participants With Clinically Significant Changes in Physical Examinations: Inhibitor and Non-Inhibitor Cohort
Physical examination included assessments of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. Height and weight were also measured and recorded. Clinical significance in physical examinations was determined by investigator.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Clinically Significant Changes in Vital Signs: Inhibitor and Non-Inhibitor Cohort
Vital signs included temperature, pulse rate, respiratory rate, and blood pressure. Blood pressure and pulse rate were measured in a supine position using completely automated device after at least 5 minutes of rest for the participant in a quiet setting without distractions. Respiratory rate was measured by observing and counting the respirations of the participant for 30 seconds and multiplied by 2. Clinical significance of vital signs was determined by investigator.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters: Inhibitor and Non-Inhibitor Cohort
Hematology included: hemoglobin; leukocytes; neutrophils; and platelets. Chemistry included: potassium; aspartate aminotransferase; creatinine; alkaline phosphatase; total bilirubin; albumin; calcium corrected, estimated decreased; sodium; and calcium. Clinical significance of laboratory parameters was determined by investigator.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Severe/ Systematic Hypersensitivity and Anaphylactic Reactions: Inhibitor and Non-Inhibitor Cohort
Systemic hypersensitivity is a complex immune response where the immune system reacts excessively to an antigen, often leading to severe allergic reactions, including widespread symptoms (which may affect multiple organs), such as rash, swelling of your face, lips, mouth, or tongue, trouble breathing, wheezing, dizziness, fainting, fast heartbeat, pounding in your chest or sweating. Anaphylaxis is a severe, potentially fatal, systemic allergic reaction that occurs suddenly after contact with an allergy causing substance.
Time frame: During prophylaxis treatment in ATP (12 months)
Model-Based ABR of Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of the following: pain or an unusual sensation in the joint, palpable swelling, and warmth of the skin over the joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Spontaneous Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as the number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, the days on treatment ended at the last dosing date + 6 days. Spontaneous bleed: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Target Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Target joint: a major joint into which repeated bleeds occurred. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of following: pain or unusual sensation in joint, palpable swelling, and warmth of the skin over joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Total Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication). Untreated bleed: If a bleed was untreated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication), it was considered as an untreated bleed. Total bleeds: Treated bleeds + untreated bleeds. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Time frame: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Change From Baseline in Hemophilia Joint Health Score (HJHS) at Month 6: Non-Inhibitor Cohort
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling \[0-3\], duration of swelling \[0-1\], muscle atrophy \[0-2\], crepitus on motion \[0-2\], flexion loss \[0-3\], extension loss \[0-3\], joint pain \[0-2\], and strength \[0-4\]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in HJHS at Month 6: Inhibitor Cohort
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling \[0-3\], duration of swelling \[0-1\], muscle atrophy \[0-2\], crepitus on motion \[0-2\], flexion loss \[0-3\], extension loss \[0-3\], joint pain \[0-2\], and strength \[0-4\]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) Total Score and Physical Health Domain at Month 6: Non-Inhibitor Cohort, Participants >=17 Years
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (\>=17 years of age) with hemophilia. It contained 46 items with 10 domains that assessed health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with haemophilia; treatment; future; family planning; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Haem-A-QoL Total Score and Physical Health Domain at Month 6: Inhibitor Cohort, Participants >=17 Years
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (\>=17 years of age) with hemophilia. It contained 46 items with 10 domains that assessed health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with haemophilia; treatment; future; family planning; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Quality of Life Questionnaire for Children (Haemo-QoL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years
Haemo-QoL assessed health-related QoL in adolescent participants (12 to \<17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Haemo-QoL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years
Haemo-QoL assessed health-related QoL in adolescent participants (12 to \<17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Activities List (HAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants >=17 Years
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (\>=17 years of age). The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in HAL Total Score at Month 6: Inhibitor Cohort, Participants >=17 Years
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (\>=17 years of age). The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Pediatric Hemophilia Activities List (pedHAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to \<17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in pedHAL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to \<17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Patient Global Impression of Change-Hemophilia (PGIC-H) at Month 6: Non-Inhibitor Cohort
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
Time frame: Month 6
PGIC-H at Month 6: Inhibitor Cohort
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
Time frame: Month 6
Change From Baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L) Index and VAS Scores at Month 6: Non-Inhibitor Cohort
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in EQ-5D-5L Index and VAS Scores at Month 6: Inhibitor Cohort
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
Time frame: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
A total of 188 participants were enrolled in the study: 128 in non-inhibitor cohort (no history of inhibitors) and 60 in inhibitor cohort (current/documented history of inhibitor). The study consisted of observational phase (OP) and active treatment phase (ATP) \[participants received study drug PF-06741086 (marstacimab)\]. Participants receiving prior on-demand (OD) therapy or prior routine prophylaxis (RP) therapy in either non-inhibitor cohort or inhibitor cohort were enrolled into OP.
| Milestone | Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP |
|---|---|---|---|---|
| Started | 3 | 37 | 57 | 91 |
| Completed | 3 | 34 | 50 | 84 |
| Not completed | 0 | 3 | 7 | 7 |
| Withdrew: Withdrawal by subject | 0 | 0 | 2 | 0 |
| Withdrew: Adverse event | 0 | 0 | 1 | 0 |
| Withdrew: Other | 0 | 1 | 0 | 0 |
| Withdrew: No longer met eligibility criteria | 0 | 0 | 0 | 5 |
| Withdrew: Protocol violation | 0 | 2 | 4 | 2 |
| Milestone | Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP |
|---|---|---|---|---|
| Started | 3 | 33 | 48 | 83 |
| Completed | 3 | 33 | 45 | 78 |
| Not completed | 0 | 0 | 3 | 5 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 4 |
| Withdrew: Adverse event | 0 | 0 | 1 | 1 |
| Withdrew: Lost to follow-up | 0 | 0 | 1 | 0 |
| Withdrew: Protocol violation | 0 | 0 | 1 | 0 |
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Model-Based Annualized Bleeding Rate (ABR) of Treated Bleeding Events: Inhibitor Cohort (Participants With Prior OD Therapy at OP) | 19.78 (16.12 to 24.27) | 1.39 (0.85 to 2.29) |
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With Prior OD Therapy at OP) | 39.86 (33.05 to 48.07) | 3.2 (2.1 to 4.88) |
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|
| Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With RP at OP) | 7.9 (5.14 to 10.66) | 5.09 (3.4 to 6.78) |
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious AEs and all other AEs.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs): Inhibitor and Non-Inhibitor Cohort | 18 | 25 | 20 | 66 | 19 | 35 | 2 | 3 |
AE: any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE: any untoward medical occurrence at any dose that resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Serious Adverse Events (SAEs): Inhibitor and Non-Inhibitor Cohort | 1 | 2 | 0 | 7 | 5 | 1 | 0 | 0 |
Thrombotic events are when a blood clot (thrombus) forms in a blood vessel in the arm, leg, lung, or head and can be life-threatening. A thrombus can occur in veins (venous thrombosis) or arteries (arterial thrombosis).
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Thrombotic Events: Inhibitor and Non-Inhibitor Cohort | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Thrombotic microangiopathy: pathological state where micro-vessels are occluded by platelet rich thrombi leading to thrombocytopenia (low platelets) and microangiopathic haemolytic anaemia (red blood cell destruction) and potential end organ damage.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Thrombotic Microangiopathy: Inhibitor and Non-Inhibitor Cohort | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Disseminated intravascular coagulation is characterized by widespread clotting in small blood vessels, and consumption of platelets and clotting factors by the clots, leading to bleeding.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: RP at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Disseminated Intravascular Coagulation/ Consumption Coagulopathy: Inhibitor and Non-Inhibitor Cohort | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
ADA positive: A participant with \>=1 treatment induced or treatment-boosted ADA response. NAb positive: An ADA positive participant with \>=1 treatment induced or treatment boosted NAb response.
| Participants | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| ADA | 10 | 13 | 10 | 0 |
| NAb | 1 | 5 | 2 | — |
Persistent ADA: participant with treatment-induced or treatment-boosted ADA detected at 2 or more times during treatment including any follow-up, where first and last ADA positive samples separated by \>=16 weeks. Transient ADA: treatment-induced or treatment-boosted ADA detected at only 1 time during treatment or follow-up. Persistent NAb: NAb-positive participant with first and last positive NAb samples detected \>=16 weeks posttreatment, irrespective of any negative samples in between. Transient NAb: NAb-positive participant with (1) treatment-induced/treatment-boosted NAb sample detected at only 1 time posttreatment or (2) treatment-induced/treatment-boosted NAb samples detected at 2 or more times where first and last positive samples separated by \<16 weeks, and participant's last sample was NAb or ADA negative.
| Participants | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| ADA — Persistent | 4 | 5 | 1 | — |
| ADA — Transient | 6 | 8 | 9 | — |
| NAb — Persistent | 0 | 0 | 1 | — |
| NAb — Transient | 1 | 5 | 1 | — |
ISR included: injection site haematoma, injection site pain, injection site bruising, injection site erythema, injection site induration, injection site oedema, Injection site pruritus and Injection site swelling. ISR graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 where grade 1: tenderness with or without associated symptoms (warmth, erythema, itching), grade 2: pain, lipodystrophy, edema, phlebitis, grade 3: ulceration or necrosis, severe tissue damage, operative intervention indicated, grade 4: life-threatening consequences, urgent intervention indicated and grade 5: death. In this outcome measure only categories with non-zero values reported.
| Participants | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Grade 1 | 2 | 8 | 3 | 0 |
| Grade 2 | 0 | 1 | 1 | 0 |
Physical examination included assessments of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. Height and weight were also measured and recorded. Clinical significance in physical examinations was determined by investigator.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: RP at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Clinically Significant Changes in Physical Examinations: Inhibitor and Non-Inhibitor Cohort | 0 | 2 | 0 | 2 | 0 | 0 | 0 | 0 |
Vital signs included temperature, pulse rate, respiratory rate, and blood pressure. Blood pressure and pulse rate were measured in a supine position using completely automated device after at least 5 minutes of rest for the participant in a quiet setting without distractions. Respiratory rate was measured by observing and counting the respirations of the participant for 30 seconds and multiplied by 2. Clinical significance of vital signs was determined by investigator.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: RP at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Clinically Significant Changes in Vital Signs: Inhibitor and Non-Inhibitor Cohort | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Hematology included: hemoglobin; leukocytes; neutrophils; and platelets. Chemistry included: potassium; aspartate aminotransferase; creatinine; alkaline phosphatase; total bilirubin; albumin; calcium corrected, estimated decreased; sodium; and calcium. Clinical significance of laboratory parameters was determined by investigator.
| Participants | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: RP at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters: Inhibitor and Non-Inhibitor Cohort | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Systemic hypersensitivity is a complex immune response where the immune system reacts excessively to an antigen, often leading to severe allergic reactions, including widespread symptoms (which may affect multiple organs), such as rash, swelling of your face, lips, mouth, or tongue, trouble breathing, wheezing, dizziness, fainting, fast heartbeat, pounding in your chest or sweating. Anaphylaxis is a severe, potentially fatal, systemic allergic reaction that occurs suddenly after contact with an allergy causing substance.
| Participants | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Number of Participants With Severe/ Systematic Hypersensitivity and Anaphylactic Reactions: Inhibitor and Non-Inhibitor Cohort | 0 | 0 | 0 | 0 |
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of the following: pain or an unusual sensation in the joint, palpable swelling, and warmth of the skin over the joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|---|---|---|---|
| Model-Based ABR of Joint Bleeds: Inhibitor and Non-Inhibitor Cohort | 34.52 (27.84 to 42.79) | 5.69 (3.36 to 8.02) | 2.85 (1.82 to 4.46) | 4.13 (2.59 to 5.67) | 15.15 (11.87 to 19.34) | 1.1 (0.59 to 2.04) |
ABR: number of bleeding episodes per year. ABR was calculated as the number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, the days on treatment ended at the last dosing date + 6 days. Spontaneous bleed: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|---|---|---|---|
| Model-Based ABR of Spontaneous Bleeds: Inhibitor and Non-Inhibitor Cohort | 32.63 (25.79 to 41.28) | 5.89 (3.57 to 8.22) | 2.45 (1.62 to 3.72) | 3.78 (2.25 to 5.31) | 15.27 (12.07 to 19.31) | 0.87 (0.53 to 1.43) |
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Target joint: a major joint into which repeated bleeds occurred. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of following: pain or unusual sensation in joint, palpable swelling, and warmth of the skin over joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|---|---|---|---|
| Model-Based ABR of Target Joint Bleeds: Inhibitor and Non-Inhibitor Cohort | 24.38 (18.27 to 32.35) | 3.37 (1.6 to 5.15) | 1.84 (1.07 to 3.18) | 2.51 (1.26 to 3.76) | 6.3 (4.32 to 9.2) | 0.79 (0.36 to 1.74) |
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication). Untreated bleed: If a bleed was untreated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication), it was considered as an untreated bleed. Total bleeds: Treated bleeds + untreated bleeds. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
| Bleeds per year | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|---|---|---|---|
| Model-Based ABR of Total Bleeds: Inhibitor and Non-Inhibitor Cohort | 49.97 (42.09 to 59.32) | 8.9 (6.02 to 11.77) | 7.41 (5.1 to 10.75) | 5.98 (4.14 to 7.82) | 27.29 (22.54 to 33.03) | 4.36 (2.65 to 7.18) |
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling \[0-3\], duration of swelling \[0-1\], muscle atrophy \[0-2\], crepitus on motion \[0-2\], flexion loss \[0-3\], extension loss \[0-3\], joint pain \[0-2\], and strength \[0-4\]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Change From Baseline in Hemophilia Joint Health Score (HJHS) at Month 6: Non-Inhibitor Cohort | -2.6 (-5.7 to 0.5) | 1.3 (-0.7 to 3.3) | -5.2 (-8.7 to 1.8) | -0.6 (-2.2 to 1.0) |
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling \[0-3\], duration of swelling \[0-1\], muscle atrophy \[0-2\], crepitus on motion \[0-2\], flexion loss \[0-3\], extension loss \[0-3\], joint pain \[0-2\], and strength \[0-4\]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Change From Baseline in HJHS at Month 6: Inhibitor Cohort | -1.1 (-4.0 to 1.9) | -3.9 (-6.1 to -1.7) |
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (\>=17 years of age) with hemophilia. It contained 46 items with 10 domains that assessed health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with haemophilia; treatment; future; family planning; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Total Score | -1.5 (-5.5 to 2.5) | -1.2 (-3.5 to 1.1) | -4.8 (-7.9 to -1.7) | -3.7 (-6.8 to -0.6) |
| Physical Health Score | -1.1 (-12.2 to 10.0) | -3.0 (-8.2 to 2.2) | -12.4 (-19.6 to -5.1) | -6.1 (-12.6 to 0.4) |
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (\>=17 years of age) with hemophilia. It contained 46 items with 10 domains that assessed health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with haemophilia; treatment; future; family planning; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Total Score | 1.5 (-2.4 to 5.5) | -12.0 (-18.3 to -5.7) |
| Physical Health Score | 2.2 (-6.6 to 11.0) | -20.8 (-29.7 to -11.9) |
Haemo-QoL assessed health-related QoL in adolescent participants (12 to \<17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Change From Baseline in Hemophilia Quality of Life Questionnaire for Children (Haemo-QoL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years | NA (NA to NA) | 0.8 (-6.2 to 7.8) | NA (NA to NA) | -5.0 (-10.5 to 0.4) |
Haemo-QoL assessed health-related QoL in adolescent participants (12 to \<17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Change From Baseline in Haemo-QoL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years | -0.1 (-9.1 to 9.0) | -7.9 (-13.7 to -2.2) |
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (\>=17 years of age). The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Change From Baseline in Hemophilia Activities List (HAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants >=17 Years | 2.0 (-1.3 to 5.2) | -0.6 (-3.0 to 1.8) | 1.1 (-2.8 to 5.1) | 2.1 (-1.0 to 5.1) |
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (\>=17 years of age). The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Change From Baseline in HAL Total Score at Month 6: Inhibitor Cohort, Participants >=17 Years | 0.4 (-5.2 to 5.9) | 10.6 (4.8 to 16.4) |
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to \<17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Change From Baseline in Pediatric Hemophilia Activities List (pedHAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years | NA (NA to NA) | -1.7 (-9.8 to 6.3) | NA (NA to NA) | 5.3 (-4.7 to 15.3) |
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to \<17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| Change From Baseline in pedHAL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years | -4.4 (-20.9 to 12.1) | 14.4 (1.1 to 27.8) |
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| Patient Global Impression of Change-Hemophilia (PGIC-H) at Month 6: Non-Inhibitor Cohort | 3.0 ± 1.35 | 4.0 ± 1.24 | 2.0 ± 1.03 | 1.0 ± 1.13 |
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| PGIC-H at Month 6: Inhibitor Cohort | 3.0 ± 1.39 | 1.0 ± 0.81 |
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
| Units on a scale | Non-Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] |
|---|---|---|---|---|
| EQ-5D-5L index score | -0.0116 (-0.0799 to 0.0566) | 0.03 (-0.014 to 0.074) | 0.0122 (-0.0627 to 0.0872) | 0.0752 (0.0178 to 0.1325) |
| EQ-VAS score | -0.2 (-6.7 to 6.2) | 3.0 (-0.6 to 6.6) | 4.2 (-0.1 to 8.5) | 4.5 (1.4 to 7.7) |
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
| Units on a scale | Inhibitor Cohort: OD at OP | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|
| EQ-5D-5L index score | 0.0026 (-0.0669 to 0.0721) | 0.1687 (0.0718 to 0.2657) |
| EQ-VAS score | 1.5 (-6.2 to 9.3) | 10.2 (4.2 to 16.2) |
Collected over OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | 0/48 (0%) | 1/48 (2.1%) | 27/48 (56.3%) |
| Inhibitor Cohort: OD at OP | 0/57 (0%) | 5/57 (8.8%) | 9/57 (15.8%) |
| Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | 0/83 (0%) | 7/83 (8.4%) | 31/83 (37.3%) |
| Non-Inhibitor Cohort: OD at OP | 0/37 (0%) | 1/37 (2.7%) | 3/37 (8.1%) |
| Inhibitor Cohort: RP at OP | 0/3 (0%) | 0/3 (0%) | 2/3 (66.7%) |
| Non-Inhibitor Cohort: RP at OP | 0/91 (0%) | 2/91 (2.2%) | 4/91 (4.4%) |
| Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | 0/33 (0%) | 0/33 (0%) | 9/33 (27.3%) |
| Event | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Non-Inhibitor Cohort: OD at OP | Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|---|---|---|---|---|---|
| Gastric haemorrhageGastrointestinal disorders | 0/3 | 0/48 | 0/57 | 0/83 | 1/37 | 0/3 | 0/91 | 0/33 |
| RashSkin and subcutaneous tissue disorders | 0/3 | 1/48 | 0/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| VomitingGastrointestinal disorders | 0/3 | 0/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Device related infectionInfections and infestations | 0/3 | 0/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Traumatic haemorrhageInjury, poisoning and procedural complications | 0/3 | 0/48 | 1/57 | 1/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| HaemarthrosisMusculoskeletal and connective tissue disorders | 0/3 | 0/48 | 1/57 | 1/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Muscle haemorrhageMusculoskeletal and connective tissue disorders | 0/3 | 0/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Coronary artery diseaseCardiac disorders | 0/3 | 0/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| HaematuriaRenal and urinary disorders | 0/3 | 0/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Condition aggravatedGeneral disorders | 0/3 | 0/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Event | Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] | Inhibitor Cohort: OD at OP | Non-Inhibitor Cohort: RP at OP/ PF-06741086 in ATP [RP at OP] | Non-Inhibitor Cohort: OD at OP | Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: RP at OP | Non-Inhibitor Cohort: OD at OP/ PF-06741086 in ATP [OD at OP] |
|---|---|---|---|---|---|---|---|---|
| Dental cariesGastrointestinal disorders | 0/3 | 4/48 | 0/57 | 4/83 | 2/37 | 1/3 | 0/91 | 0/33 |
| Fibrin D dimer increasedInvestigations | 1/3 | 4/48 | 3/57 | 3/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Protein urine presentInvestigations | 1/3 | 3/48 | 1/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Prothrombin fragment 1.2 increasedInvestigations | 1/3 | 0/48 | 0/57 | 3/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Blood loss anaemiaBlood and lymphatic system disorders | 0/3 | 0/48 | 0/57 | 0/83 | 0/37 | 1/3 | 0/91 | 0/33 |
| BronchitisInfections and infestations | 0/3 | 0/48 | 0/57 | 0/83 | 0/37 | 1/3 | 0/91 | 0/33 |
| LaryngitisInfections and infestations | 0/3 | 0/48 | 0/57 | 0/83 | 0/37 | 1/3 | 0/91 | 0/33 |
| Ligament sprainInjury, poisoning and procedural complications | 0/3 | 0/48 | 0/57 | 0/83 | 0/37 | 1/3 | 0/91 | 0/33 |
| DiarrhoeaGastrointestinal disorders | 1/3 | 0/48 | 0/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| GastroenteritisInfections and infestations | 1/3 | 0/48 | 0/57 | 0/83 | 0/37 | 0/3 | 0/91 | 0/33 |
| Age, Customized(Participants) | Non-Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Total |
|---|---|---|---|---|---|
| Age — <18 years | 18 | 2 | 15 | 1 | 36 |
| Age — Between 18 and 64 years | 72 | 35 | 40 | 2 | 149 |
| Age — >=65 years | 1 | 0 | 2 | 0 | 3 |
| Sex: Female, Male(Participants) | Non-Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Total |
|---|---|---|---|---|---|
| Female | 0 | 0 | 0 | 0 | 0 |
| Male | 91 | 37 | 57 | 3 | 188 |
| Ethnicity (NIH/OMB)(Participants) | Non-Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 9 | 4 | 2 | 0 | 15 |
| Not Hispanic or Latino | 82 | 33 | 54 | 3 | 172 |
| Unknown or Not Reported | 0 | 0 | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | Non-Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Non-Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: OD at OP + PF-06741086 in ATP | Inhibitor Cohort: Prophylaxis at OP + PF-06741086 in ATP | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Asian | 37 | 24 | 29 | 3 | 93 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 1 | 0 | 8 | 0 | 9 |
| White | 52 | 13 | 19 | 0 | 84 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 0 | 1 | 0 | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Data sharing details ↗ · www.pfizer.com
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Studies of the drug tested here: Marstacimab