A Phase 3 interventional study of PEGylated Recombinant Factor VIII and ITI in Hemophilia A, sponsored by Baxalta now part of Shire. Completed at 91 sites in 23 countries. Open to participants aged Up to 5 Years. Per ClinicalTrials.gov, last updated 2025-07-28.
Sponsored by Baxalta now part of Shire (part of Takeda) · Phase 3, Interventional, and Treatment
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates.
The main aim of the study is to check for side effects from treatment with BAX855. This includes the buildup of antibodies against FVIII which may stop BAX855 from working properly. Another aim is to learn how well BAX855 controls bleeding.
In this study, the children can receive BAX855 either as preventative treatment (prophylaxis), or as needed to treat bleeding (on-demand).
In case a participant develops antibodies, treatment will be provided as part of the study.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 120 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →Baxalta now part of Shire is the lead sponsor of 111 studies on the registry; none are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.
First submitted before Baxalta became part of Takeda.
Counted across the registry records on this site, refreshed daily.
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.
Additional inclusion criteria for Part B (immune tolerance induction [ITI]).
Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (greater than or equal to [>=] 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with
Exclusion Criteria
Additional exclusion criteria for Part B (ITI)
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.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
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.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
Previously Untreated Patients (PUPs) \< 6 years of age with severe hemophilia A (FVIII \< 1%) and \< 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion were enrolled in a single arm group. Part A (Main Study): Participants age \<3 years - who had not experienced two joint bleeds received on-demand treatment of 10-80 international units per kilogram (IU/kg) intravenously (IV) depending on the severity of the bleeding episode; and - who experienced maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part B (Immune tolerance induction \[ITI\] Portion): Participants who met the pre-defined Part B treatment criteria entered Part B of the study for ITI. Participants either received prophylaxis treatment of BAX 855 low dose 50 IU/kg IV, three times a week or high dose 100-200 IU/kg IV, daily at discretion of the investigator according to the institution's standard of care.
Biological: PEGylated Recombinant Factor VIII · Biological: ITI
Polyethylene glycol (PEG)-ylated full-length recombinant FVIII (rFVIII).
Also known as: ADYNOVATE, BAX 855, TAK-660
Immune tolerance induction therapy
Number of Participants With FVIII Inhibitor Development
Number of participants who developed an inhibitor (at any time) confirmed by a central laboratory based on a second repeat blood sample draw within 2 weeks of site notification of an inhibitor and all participants who had not developed an inhibitor and had greater than or equal to (\>=) 100 EDs when the sample for the last valid inhibitor test was drawn.
Time frame: Throughout Part A of the study, approximately 5 years
Number of Participants With Success of Immune Tolerance Induction (ITI)
Success is defined as 1) a persistently negative inhibitor titer less than (\<) 0.6 Bethesda unit (BU), 2) FVIII IR \>=66% of the baseline value following a wash-out period of 84-96 hours, and 3) a FVIII half-life of \>=6 hours.
Time frame: Up to 33 months in Part B of the study
Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies
Binding IgG and IgM antibodies to FVIII , Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) was assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1(Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) and Study Completion Visit (Weeks 100-110).
Time frame: Throughout Part A of the study, approximately 5 years
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A SAE is any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to investigational product or not and at any dose) which results in death, is life-threatening, requires inpatient hospitalization, prolongation of hospitalization, is an important medical event. Number of participants with AEs and SAEs in both Part A and Part B were assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
Time frame: Throughout Part A and Part B of the study, approximately 9 years
Number of Participants With At Least One Clinically Significant Changes in Vital Signs
Vital signs were assessed based on body temperature, respiratory rate, blood pressure, and heart rate.
Time frame: Throughout Part A and Part B of the study, approximately 9 years
Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters
Clinical laboratory parameters included hematology and clinical chemistry. Changes in laboratory values could be considered as AE if they were judged to be clinically significant.
Time frame: Throughout Part A and Part B of the study, approximately 9 years
Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI)
ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. Bleeding occurring at multiple locations related to the same injury (example, knee and ankle bleed following a fall) was counted as a single bleeding episode. Mean total annualized bleed rate is reported. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
Time frame: Throughout Part A and Part B of the study, approximately 9 years
Bleeding Episodes Categorized by Number of BAX 855 Infusions Required for Treatment
A bleeding episode is defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The number of BAX 855 infusions needed for each bleeding episode was determined by the participant, caregiver, clinician treating the participant, and is based upon the participant's response to treatment, using the Efficacy Rating Scale for Treatment of Bleeding Episodes. Number of bleeding episodes are categorized by number of infusions required to treat the bleeding episodes. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
Time frame: Throughout Part A of the study, approximately 5 years
Number of Bleeds by Overall Hemostatic Efficacy Rating at 24 Hours After Initiation of Treatment
The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain \& cessation of objective signs of bleeding after single infusion \& no additional infusion is required for the control of bleeding; Good:Definite pain relief \&/or improvement in signs of bleeding after single infusion \& possibly requires more than 1 infusion for complete resolution; Fair:Probable \&/or slight relief of pain \& slight improvement in signs of bleeding after single infusion \& required more than 1 infusion for complete resolution \& None:No improvement or condition worsens.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first \& then moved to prophylaxis treatment was counted for both on-demand \& prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen.
Time frame: At 24 hours after study drug administration during Part A of the study
Number of Bleeds by Overall Hemostatic Efficacy Rating at Bleed Resolution
The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain \& cessation of objective signs of bleeding after single infusion \& no additional infusion is required for the control of bleeding; Good:Definite pain relief \&/or improvement in signs of bleeding after a single infusion \& possibly requires more than 1 infusion for complete resolution; Fair:Probable \&/or slight relief of pain \& slight improvement in signs of bleeding after single infusion \& required more than 1 infusion for complete resolution \& None:No improvement or condition worse.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first \& then moved to prophylaxis treatment was counted for both on-demand \& prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen.
Time frame: From start of study treatment up to bleed resolution throughout Part A of the study (up to approximately 5 years)
Weight-adjusted Consumption of BAX 855: Average Prophylactic Dose
Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose per prophylactic infusion, per month and per year are reported as categories.
Time frame: Throughout Part A of the study, approximately 5 years
Weight-adjusted Consumption of BAX 855: Average Number of Prophylactic Infusions
Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual number of BAX 855 infusions as measured in the clinic. Average number of infusions per month and year are reported as categories.
Time frame: Throughout Part A and Part B of the study, approximately 9 years
Weight-adjusted Consumption of BAX 855: Average Dose
Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose to treat bleeding episode and average FVIII inhibitor treatment Dose \[IU/kg\] per Week, Month and per Year are reported as categories. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
Time frame: Throughout Part A of the study, approximately 5 years
Number of Participants by Hemostatic Efficacy Rating in Case of Surgery
Hemostatic efficacy was assessed during \& after any surgical or invasive procedures,\& overall as a perioperative assessment.Operating surgeon assessed hemostatic efficacy compared to that expected for the type of procedure performed in non-hemophilic population,prior to discharge from recovery room(intraoperative),on postoperative Day 1 \& at discharge or 14 days post-surgery(perioperative).Participants rated efficacy using following ratings:1.Excellent:Postoperative blood loss was ≤100% than expected;2.Good:Postoperative blood loss was up to 50% more (101-150%) than expected;3.Fair:Postoperative blood loss was more than 50% (\>150%) of that expected;4.None:Significant postoperative bleeding that was result of inadequate therapeutic response despite proper dosing,necessitating rescue therapy.Perioperative ratings also considered amount of blood components required for transfusions compared to expected.Participant-provided ratings for each of the assessments are reported as categories.
Time frame: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)
Blood Loss Per Participant in Case of Surgery
The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Post-operatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. The assessment was done for the intra-operative time period (prior to discharge from recovery room) and for the post-operative time period (from completion of the procedure until approximately 24 hours post-surgery).
Time frame: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)
Incremental Recovery (IR) of BAX 855
BAX 855 was administered in participants for the determination of FVIII IR at study site at baseline \& every study visit other than study visits at 5 EDs, 15 EDs \& 30 EDs. FVIII assays were done using following methods:1-stage clotting FVIII activity \& FVIII chromogenic activity. Data is reported for each of these methods as categories per visit. Study Completion assessment was conducted at end of the Main Study \& again at end of the ITI portion. Thus, more number of participants were analyzed at study completion visit than those who actually completed the overall study. IR is reported as a ratio of (IU/deciliter \[dL\])/(IU/kg), calculated as: IR = (Cmax- (C pre-infusion)) / (IU/kg), where C=concentration. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1 (Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) \& Study Completion Visit (Weeks 100-110).
Time frame: Baseline up to Study Completion (Up to 5 years in Part A and up to 3.5 years in Part B)
Half-life (T1/2) of BAX 855
The Half-life to determine FVIII half-life was an optional assessment that was planned to be performed at baseline, Visit 1, or Visit 2.
Time frame: Pre-infusion, Post-infusion: 15-30 minutes and 24-48 hours at Baseline
Immune Tolerance Induction (ITI) - Number of Participants With Partial Success and Failure of ITI
Partial success defined as which meet two of following criteria, 1) inhibitor titer \<0.6 BU (confirmed by a central laboratory with a second blood specimen obtained within 2 months), 2) FVIII in vivo recovery \>=66% of baseline value (confirmed within a two month period), and 3) FVIII half-life \>=6 hours. Failure defined as the failure to meet the criteria for partial success.
Time frame: Up to 33 months in Part B of the study
Immune Tolerance Induction (ITI) - Number of Participants With At Least One Catheter-related Complication
Number of participants with catheter-related complications are reported.
Time frame: Up to 33 months in Part B of the study
Immune Tolerance Induction (ITI) - Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies
Binding IgG and IgM antibodies to Factor VIII (FVIII), Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) are reported as categories per visit.
Time frame: Up to 33 months in Part B of the study
Participants took part in the study at various investigative sites globally from 12 November 2015 to 29 October 2024.
| Milestone | All Participants |
|---|---|
| Started | 120 |
| Part a: on-demand treatment | 80 |
| Part a: prophylaxis treatment | 112 |
| Completed | 106 |
| Not completed | 14 |
| Withdrew: Adverse event | 3 |
| Withdrew: Physician decision | 2 |
| Withdrew: Withdrawal by subject | 7 |
| Withdrew: Reason not specified | 2 |
| Milestone | All Participants |
|---|---|
| Started | 7 |
| Part b:fvii inhibitor treatment(50iu/kg 3 times weekly regimen) | 4 |
| Part b:fvii inhibitor treatment (100-200iu/kg daily regimen) | 3 |
| Completed | 7 |
| Not completed | 0 |
Number of participants who developed an inhibitor (at any time) confirmed by a central laboratory based on a second repeat blood sample draw within 2 weeks of site notification of an inhibitor and all participants who had not developed an inhibitor and had greater than or equal to (\>=) 100 EDs when the sample for the last valid inhibitor test was drawn.
| Participants | Part A: Main Study |
|---|---|
| Number of Participants With FVIII Inhibitor Development | 11 |
Success is defined as 1) a persistently negative inhibitor titer less than (\<) 0.6 Bethesda unit (BU), 2) FVIII IR \>=66% of the baseline value following a wash-out period of 84-96 hours, and 3) a FVIII half-life of \>=6 hours.
| Participants | Part B: ITI Portion |
|---|---|
| Number of Participants With Success of Immune Tolerance Induction (ITI) | 5 |
Binding IgG and IgM antibodies to FVIII , Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) was assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1(Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) and Study Completion Visit (Weeks 100-110).
| Participants | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis |
|---|---|---|
| Screening- Binding IgG Antibodies to FVIII | 1 | 0 |
| Screening- Binding IgM Antibodies to FVIII | 0 | 0 |
| Screening- Binding IgG Antibodies to PEG-FVIII | 3 | 2 |
| Screening- Binding IgM Antibodies to PEG-FVIII | 4 | 1 |
| Screening- Binding IgG Antibodies to PEG | 0 | 0 |
| Screening- Binding IgM Antibodies to PEG | 6 | 4 |
| Baseline- Binding IgG Antibodies to FVIII | 2 | 1 |
| Baseline- Binding IgM Antibodies to FVIII | 0 | 0 |
| Baseline- Binding IgG Antibodies to PEG-FVIII | 7 | 3 |
| Baseline- Binding IgM Antibodies to PEG-FVIII | 4 | 2 |
| Baseline- Binding IgG Antibodies to PEG | 0 | 0 |
| Baseline- Binding IgM Antibodies to PEG | 8 | 2 |
| Visit 1- Binding IgG Antibodies to FVIII | 0 | 4 |
| Visit 1- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 1- Binding IgG Antibodies to PEG-FVIII | 14 | 36 |
| Visit 1- Binding IgM Antibodies to PEG-FVIII | 0 | 2 |
| Visit 1- Binding IgG Antibodies to PEG | 4 | 8 |
| Visit 1- Binding IgM Antibodies to PEG | 0 | 3 |
| Visit 2- Binding IgG Antibodies to FVIII | 1 | 1 |
| Visit 2- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 2- Binding IgG Antibodies to PEG-FVIII | 6 | 27 |
| Visit 2- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 2- Binding IgG Antibodies to PEG | 2 | 3 |
| Visit 2- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 3- Binding IgG Antibodies to FVIII | 0 | 2 |
| Visit 3- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 3- Binding IgG Antibodies to PEG-FVIII | 4 | 25 |
| Visit 3- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 3- Binding IgG Antibodies to PEG | 1 | 4 |
| Visit 3- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 4- Binding IgG Antibodies to FVIII | 0 | 0 |
| Visit 4- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 4- Binding IgG Antibodies to PEG-FVIII | 1 | 18 |
| Visit 4- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 4- Binding IgG Antibodies to PEG | 0 | 1 |
| Visit 4- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 5- Binding IgG Antibodies to FVIII | 0 | 0 |
| Visit 5- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 5- Binding IgG Antibodies to PEG-FVIII | 1 | 13 |
| Visit 5- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 5- Binding IgG Antibodies to PEG | 0 | 1 |
| Visit 5- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 6- Binding IgG Antibodies to FVIII | 0 | 0 |
| Visit 6- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 6- Binding IgG Antibodies to PEG-FVIII | 1 | 5 |
| Visit 6- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 6- Binding IgG Antibodies to PEG | 0 | 0 |
| Visit 6- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 7- Binding IgG Antibodies to FVIII | 0 | 0 |
| Visit 7- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 7- Binding IgG Antibodies to PEG-FVIII | 1 | 4 |
| Visit 7- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 7- Binding IgG Antibodies to PEG | 0 | 0 |
| Visit 7- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 8- Binding IgG Antibodies to FVIII | 0 | 0 |
| Visit 8- Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 8- Binding IgG Antibodies to PEG-FVIII | 0 | 1 |
| Visit 8- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 8- Binding IgG Antibodies to PEG | 0 | 0 |
| Visit 8- Binding IgM Antibodies to PEG | 0 | 0 |
| Study Completion- Binding IgG Antibodies to FVIII | 0 | 1 |
| Study Completion- Binding IgM Antibodies to FVIII | 0 | 0 |
| Study Completion-Binding IgG Antibodies to PEG-FVIII | 1 | 2 |
| Study Completion- Binding IgM Antibodies to PEG-FVIII | 1 | 0 |
| Study Completion- Binding IgG Antibodies to PEG | 1 | 1 |
| Study Completion- Binding IgM Antibodies to PEG | 1 | 0 |
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A SAE is any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to investigational product or not and at any dose) which results in death, is life-threatening, requires inpatient hospitalization, prolongation of hospitalization, is an important medical event. Number of participants with AEs and SAEs in both Part A and Part B were assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
| Participants | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|---|---|
| AEs | 57 | 94 | 4 | 3 |
| SAEs | 18 | 35 | 1 | 3 |
Vital signs were assessed based on body temperature, respiratory rate, blood pressure, and heart rate.
| Participants | Part A: Main Study | Part B: ITI Portion |
|---|---|---|
| Number of Participants With At Least One Clinically Significant Changes in Vital Signs | 0 | 0 |
Clinical laboratory parameters included hematology and clinical chemistry. Changes in laboratory values could be considered as AE if they were judged to be clinically significant.
| Participants | Part A: Main Study | Part B: ITI Portion |
|---|---|---|
| Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters | 53 | 1 |
ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. Bleeding occurring at multiple locations related to the same injury (example, knee and ankle bleed following a fall) was counted as a single bleeding episode. Mean total annualized bleed rate is reported. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
| unique bleeds per year | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|---|---|
| Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI) | 10.004 ± 15.533 | 4.536 ± 8.657 | 7.673 ± 8.965 | 6.432 ± 14.518 |
A bleeding episode is defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The number of BAX 855 infusions needed for each bleeding episode was determined by the participant, caregiver, clinician treating the participant, and is based upon the participant's response to treatment, using the Efficacy Rating Scale for Treatment of Bleeding Episodes. Number of bleeding episodes are categorized by number of infusions required to treat the bleeding episodes. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
| bleeding episodes | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis |
|---|---|---|
| Number of infusions per bleed: 0 | 7 | 14 |
| Number of infusions per bleed: 1 | 302 | 312 |
| Number of infusions per bleed: 2 | 43 | 44 |
| Number of infusions per bleed: 3 | 18 | 19 |
| Number of infusions per bleed: 4 | 4 | 1 |
| Number of infusions per bleed: >4 | 5 | 6 |
The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain \& cessation of objective signs of bleeding after single infusion \& no additional infusion is required for the control of bleeding; Good:Definite pain relief \&/or improvement in signs of bleeding after single infusion \& possibly requires more than 1 infusion for complete resolution; Fair:Probable \&/or slight relief of pain \& slight improvement in signs of bleeding after single infusion \& required more than 1 infusion for complete resolution \& None:No improvement or condition worsens.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first \& then moved to prophylaxis treatment was counted for both on-demand \& prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen.
| bleeds | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis |
|---|---|---|
| Excellent | 76 | 102 |
| Good | 67 | 71 |
| Fair | 13 | 10 |
| None | 2 | 1 |
The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain \& cessation of objective signs of bleeding after single infusion \& no additional infusion is required for the control of bleeding; Good:Definite pain relief \&/or improvement in signs of bleeding after a single infusion \& possibly requires more than 1 infusion for complete resolution; Fair:Probable \&/or slight relief of pain \& slight improvement in signs of bleeding after single infusion \& required more than 1 infusion for complete resolution \& None:No improvement or condition worse.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first \& then moved to prophylaxis treatment was counted for both on-demand \& prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen.
| bleeds | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis |
|---|---|---|
| Excellent | 166 | 166 |
| Good | 83 | 77 |
| Fair | 12 | 9 |
| None | 3 | 1 |
Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose per prophylactic infusion, per month and per year are reported as categories.
| IU/kg | Part A: Main Study: Prophylaxis |
|---|---|
| Average Dose [IU/kg] per Prophylactic Infusion | 45.514 ± 7.831 |
| Average Prophylactic Dose [IU/kg] per Month | 221.568 ± 85.925 |
| Average Prophylactic Dose [IU/kg] per Year | 2597.079 ± 783.089 |
Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual number of BAX 855 infusions as measured in the clinic. Average number of infusions per month and year are reported as categories.
| infusions | Part A: Main Study: Prophylaxis |
|---|---|
| Average Number of Prophylactic Infusions per Month | 4.900 ± 1.877 |
| Average Number of Prophylactic Infusions per Year | 57.067 ± 16.361 |
Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose to treat bleeding episode and average FVIII inhibitor treatment Dose \[IU/kg\] per Week, Month and per Year are reported as categories. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen.
| IU/kg | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|---|---|
| Average Dose [IU/kg] to Treat Bleeding Episode | 55.445 ± 32.826 | 63.864 ± 44.818 | — | — |
| Average FVIII Inhibitor Treatment Dose [IU/kg] per Week | — | — | 123.510 ± 65.563 | 417.750 ± 319.694 |
| Average FVIII Inhibitor Treatment Dose [IU/kg] per Month | — | — | 537.038 ± 285.076 | 1816.427 ± 1390.068 |
| Average FVIII Inhibitor Treatment Dose [IU/kg] per Year | — | — | 6444.460 ± 3420.911 | 21797.128 ± 16680.821 |
Hemostatic efficacy was assessed during \& after any surgical or invasive procedures,\& overall as a perioperative assessment.Operating surgeon assessed hemostatic efficacy compared to that expected for the type of procedure performed in non-hemophilic population,prior to discharge from recovery room(intraoperative),on postoperative Day 1 \& at discharge or 14 days post-surgery(perioperative).Participants rated efficacy using following ratings:1.Excellent:Postoperative blood loss was ≤100% than expected;2.Good:Postoperative blood loss was up to 50% more (101-150%) than expected;3.Fair:Postoperative blood loss was more than 50% (\>150%) of that expected;4.None:Significant postoperative bleeding that was result of inadequate therapeutic response despite proper dosing,necessitating rescue therapy.Perioperative ratings also considered amount of blood components required for transfusions compared to expected.Participant-provided ratings for each of the assessments are reported as categories.
| Participants | All Participants |
|---|---|
| Intraoperative - Excellent | 10 |
| Intraoperative - Good | 0 |
| Intraoperative - Fair | 0 |
| Intraoperative - None | 1 |
| Postoperative - Excellent | 11 |
| Postoperative - Good | 0 |
| Postoperative - Fair | 0 |
| Postoperative - None | 0 |
| Perioperative - Excellent | 10 |
| Perioperative - Good | 0 |
| Perioperative - Fair | 0 |
| Perioperative - None | 1 |
The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Post-operatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. The assessment was done for the intra-operative time period (prior to discharge from recovery room) and for the post-operative time period (from completion of the procedure until approximately 24 hours post-surgery).
| milliliters (mL) | All Participants |
|---|---|
| Observed Intra-Operative Blood Loss | 4.8 ± 5.43 |
BAX 855 was administered in participants for the determination of FVIII IR at study site at baseline \& every study visit other than study visits at 5 EDs, 15 EDs \& 30 EDs. FVIII assays were done using following methods:1-stage clotting FVIII activity \& FVIII chromogenic activity. Data is reported for each of these methods as categories per visit. Study Completion assessment was conducted at end of the Main Study \& again at end of the ITI portion. Thus, more number of participants were analyzed at study completion visit than those who actually completed the overall study. IR is reported as a ratio of (IU/deciliter \[dL\])/(IU/kg), calculated as: IR = (Cmax- (C pre-infusion)) / (IU/kg), where C=concentration. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1 (Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) \& Study Completion Visit (Weeks 100-110).
| ratio | All Participants |
|---|---|
| Baseline-1-Stage Clotting | 1.546 ± 46.991 |
| Baseline-Chromogenic | 1.594 ± 55.555 |
| Visit 1-1-Stage Clotting | 1.452 ± 49.680 |
| Visit 1-Chromogenic | 1.651 ± 43.755 |
| Visit 2-1-Stage Clotting | 1.345 ± 72.207 |
| Visit 2-Chromogenic | 1.451 ± 57.891 |
| Visit 3-1-Stage Clotting | 1.439 ± 61.369 |
| Visit 3-Chromogenic | 1.586 ± 54.653 |
| Visit 4-1-Stage Clotting | 1.558 ± 33.938 |
| Visit 4-Chromogenic | 1.802 ± 31.897 |
| Visit 5-1-Stage Clotting | 1.588 ± 51.122 |
| Visit 5-Chromogenic | 1.916 ± 28.373 |
| Visit 6-1-Stage Clotting | 1.714 ± 34.037 |
| Visit 6-Chromogenic | 1.809 ± 46.022 |
| Visit 7-1-Stage Clotting | 1.742 ± 46.707 |
| Visit 7-Chromogenic | 1.965 ± 20.272 |
| Visit 8-1-Stage Clotting | 1.634 ± 43.470 |
| Visit 8-Chromogenic | 1.893 ± 36.334 |
| Study Completion-1-Stage Clotting | 1.819 ± 25.513 |
| Study Completion-Chromogenic | 1.979 ± 23.846 |
| Preoperative Assessments Surgery-1-Stage Clotting | 1.481 ± 42.457 |
| Preoperative Assessments Surgery-Chromogenic | 1.716 ± 45.340 |
The Half-life to determine FVIII half-life was an optional assessment that was planned to be performed at baseline, Visit 1, or Visit 2.
No measurements were reported for this outcome.
Partial success defined as which meet two of following criteria, 1) inhibitor titer \<0.6 BU (confirmed by a central laboratory with a second blood specimen obtained within 2 months), 2) FVIII in vivo recovery \>=66% of baseline value (confirmed within a two month period), and 3) FVIII half-life \>=6 hours. Failure defined as the failure to meet the criteria for partial success.
| Participants | Part B: ITI Portion |
|---|---|
| Partial Success | 1 |
| Failure | 1 |
Number of participants with catheter-related complications are reported.
| Participants | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|
| Immune Tolerance Induction (ITI) - Number of Participants With At Least One Catheter-related Complication | 0 | 2 |
Binding IgG and IgM antibodies to Factor VIII (FVIII), Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) are reported as categories per visit.
| Participants | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|
| Screening- Binding IgG Antibodies to FVIII | 0 | 1 |
| Screening- Binding IgM Antibodies to FVIII | 0 | 0 |
| Screening- Binding IgG Antibodies to PEG-FVIII | 0 | 1 |
| Screening- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Screening- Binding IgG Antibodies to PEG | 0 | 1 |
| Screening- Binding IgM Antibodies to PEG | 0 | 0 |
| First Infusion- Binding IgG Antibodies to FVIII | 2 | 0 |
| First Infusion- Binding IgM Antibodies to FVIII | 0 | 0 |
| First Infusion- Binding IgG Antibodies to PEG-FVIII | 2 | 1 |
| First Infusion- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| First Infusion- Binding IgG Antibodies to PEG | 0 | 0 |
| First Infusion- Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 1 (Week 2) - Binding IgG Antibodies to FVIII | 2 | 1 |
| Visit 1 (Week 2) - Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 1 (Week 2) - Binding IgG Antibodies to PEG-FVIII | 0 | 2 |
| Visit 1 (Week 2) - Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 1 (Week 2) - Binding IgG Antibodies to PEG | 0 | 2 |
| Visit 1 (Week 2) - Binding IgM Antibodies to PEG | 0 | 0 |
| Visit 2 (Week 4) - Binding IgG Antibodies to FVIII | 1 | 1 |
| Visit 2 (Week 4) - Binding IgM Antibodies to FVIII | 0 | 0 |
| Visit 2 (Week 4) - Binding IgG Antibodies to PEG-FVIII | 1 | 1 |
| Visit 2 (Week 4) - Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Visit 2 (Week 4) - Binding IgG Antibodies to PEG | 0 | 0 |
| Visit 2 (Week 4) - Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 1- Binding IgG Antibodies to FVIII | 1 | 0 |
| Follow-Up Visit Month 1- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 1- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 1- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 1- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 1- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 2- Binding IgG Antibodies to FVIII | 1 | 0 |
| Follow-Up Visit Month 2- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 2- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 2- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 2- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 2- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 3- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 3- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 3- Binding IgG Antibodies to PEG-FVIII | 1 | 0 |
| Follow-Up Visit Month 3- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 3- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 3- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 4- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 4-Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 4-Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 4-Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 5- Binding IgG Antibodies to FVIII | 1 | 0 |
| Follow-Up Visit Month 5- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 5- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 5- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 5- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 5- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 6- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 6- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 6- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 6- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 6- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 6- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 7-Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 7-Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 7-Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 7-Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 7-Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 7-Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 8-Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 8-Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 8-Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 8-Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 8-Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 8-Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 9-Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 9-Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 9-Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 9-Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 9-Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 9-Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 10- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 10- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 10- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 10- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 10- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 10- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 11- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 11- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 11- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 11- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 11- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 11- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 12- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 12- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 12- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 12- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 12- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 12- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 13- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 13- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 13- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 13- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 13- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 13- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 14- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 14- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 14- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 14- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 14- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 14- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 15- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 15- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 15- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 15- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 15- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 15- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 16- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 16- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 16- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 16- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 16- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 16- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 17- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 17- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 17- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 17- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 17- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 17- Binding IgM Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 18- Binding IgG Antibodies to FVIII | 0 | — |
| Follow-Up Visit Month 18- Binding IgM Antibodies to FVIII | 0 | — |
| Follow-Up Visit Month 18- Binding IgG Antibodies to PEG-FVIII | 0 | — |
| Follow-Up Visit Month 18- Binding IgM Antibodies to PEG-FVIII | 0 | — |
| Follow-Up Visit Month 18- Binding IgG Antibodies to PEG | 0 | — |
| Follow-Up Visit Month 18- Binding IgM Antibodies to PEG | 0 | — |
| Follow-Up Visit Month 19- Binding IgG Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 19- Binding IgM Antibodies to FVIII | 0 | 0 |
| Follow-Up Visit Month 19- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 19- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| Follow-Up Visit Month 19- Binding IgG Antibodies to PEG | 0 | 0 |
| Follow-Up Visit Month 19- Binding IgM Antibodies to PEG | 0 | 0 |
| FVIII Inhibitor Treatment Completion- Binding IgG Antibodies to FVIII | 0 | 0 |
| FVIII Inhibitor Treatment Completion- Binding IgM Antibodies to FVIII | 0 | 0 |
| FVIII Inhibitor Treatment Completion- Binding IgG Antibodies to PEG-FVIII | 0 | 0 |
| FVIII Inhibitor Treatment Completion- Binding IgM Antibodies to PEG-FVIII | 0 | 0 |
| FVIII Inhibitor Treatment Completion- Binding IgG Antibodies to PEG | 0 | 0 |
| FVIII Inhibitor Treatment Completion- Binding IgM Antibodies to PEG | 0 | 0 |
Collected over Up to approximately 9 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: Main Study: On-demand | 0/80 (0%) | 18/80 (22.5%) | 47/80 (58.8%) |
| Part A: Main Study: Prophylaxis | 0/112 (0%) | 35/112 (31.3%) | 77/112 (68.8%) |
| Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | 0/4 (0%) | 1/4 (25%) | 4/4 (100%) |
| Part B: ITI Portion (100-200 IU/kg Daily Regimen) | 0/3 (0%) | 3/3 (100%) | 2/3 (66.7%) |
| Event | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|---|---|
| Bacterial infectionInfections and infestations | 0/80 | 0/112 | 0/4 | 1/3 |
| Device related sepsisInfections and infestations | 0/80 | 0/112 | 0/4 | 1/3 |
| Factor VIII inhibitionBlood and lymphatic system disorders | 5/80 | 6/112 | 0/4 | 1/3 |
| PyrexiaGeneral disorders | 0/80 | 4/112 | 0/4 | 1/3 |
| TonsillitisInfections and infestations | 0/80 | 0/112 | 0/4 | 1/3 |
| Vascular device infectionInfections and infestations | 0/80 | 0/112 | 0/4 | 1/3 |
| Vascular device occlusionGeneral disorders | 0/80 | 0/112 | 0/4 | 1/3 |
| Mouth haemorrhageGastrointestinal disorders | 0/80 | 0/112 | 1/4 | 0/3 |
| Muscle haemorrhageMusculoskeletal and connective tissue disorders | 2/80 | 0/112 | 1/4 | 0/3 |
| Pharyngeal haemorrhageRespiratory, thoracic and mediastinal disorders | 0/80 | 1/112 | 1/4 | 0/3 |
| Event | Part A: Main Study: On-demand | Part A: Main Study: Prophylaxis | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Part B: ITI Portion (100-200 IU/kg Daily Regimen) |
|---|---|---|---|---|
| NasopharyngitisInfections and infestations | 6/80 | 18/112 | 2/4 | 0/3 |
| Upper respiratory tract infectionInfections and infestations | 12/80 | 27/112 | 2/4 | 0/3 |
| AnaemiaBlood and lymphatic system disorders | 0/80 | 2/112 | 0/4 | 1/3 |
| Bacillus test positiveInvestigations | 0/80 | 0/112 | 0/4 | 1/3 |
| COVID-19Infections and infestations | 1/80 | 5/112 | 0/4 | 1/3 |
| ConstipationGastrointestinal disorders | 3/80 | 2/112 | 1/4 | 1/3 |
| CoughRespiratory, thoracic and mediastinal disorders | 7/80 | 16/112 | 0/4 | 1/3 |
| Head injuryInjury, poisoning and procedural complications | 1/80 | 3/112 | 0/4 | 1/3 |
| PyrexiaGeneral disorders | 17/80 | 31/112 | 1/4 | 1/3 |
| RashSkin and subcutaneous tissue disorders | 3/80 | 3/112 | 0/4 | 1/3 |
The Safety Analysis Set (SAS) included all participants in the enrolled population with at least one BAX 855 infusion.
| Age, Continuous(years) | All Participants |
|---|---|
| Mean | 0.90 ± 0.717 |
| Sex: Female, Male(Participants) | All Participants |
|---|---|
| Female | 0 |
| Male | 120 |
| Ethnicity (NIH/OMB)(Participants) | All Participants |
|---|---|
| Hispanic or Latino | 6 |
| Not Hispanic or Latino | 114 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | All Participants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 45 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 7 |
| White | 61 |
| More than one race | 4 |
| Unknown or Not Reported | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Data sharing details ↗ · vivli.org
Ran from Nov 2015 to primary completion in Oct 2024, 107 months.
Results were first posted in Jul 2025, 8 months after primary completion.
Of 89 industry-sponsored phase 3 interventional studies under Hemophilia A, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 56. This study's 107 months is above that.
Counting every sponsor, half of 91 studies reached it within 33 months; a quarter within 20 and three quarters within 56.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: BAX 855