Phase 3
N=120
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT02615691 ↗Enrolled (actual)
120
Serious AEs
28.6%
Results posted
Jul 2025
Primary outcome: Primary: Number of Participants With FVIII Inhibitor Development — 11 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- PEGylated Recombinant Factor VIII (Biological); ITI (Biological)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Oct 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With FVIII Inhibitor Development |
11 | — |
| PRIMARY Number of Participants With Success of Immune Tolerance Induction (ITI) |
5 | — |
| SECONDARY Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies |
1; 0; 0; 0; 3; 2 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
57; 94; 4; 3; 18; 35 | — |
| SECONDARY Number of Participants With At Least One Clinically Significant Changes in Vital Signs |
0; 0 | — |
| SECONDARY Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters |
53; 1 | — |
| SECONDARY Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI) |
10.004; 4.536; 7.673; 6.432 | — |
| SECONDARY Bleeding Episodes Categorized by Number of BAX 855 Infusions Required for Treatment |
7; 14; 302; 312; 43; 44 | — |
| SECONDARY Number of Bleeds by Overall Hemostatic Efficacy Rating at 24 Hours After Initiation of Treatment |
76; 102; 67; 71; 13; 10 | — |
| SECONDARY Number of Bleeds by Overall Hemostatic Efficacy Rating at Bleed Resolution |
166; 166; 83; 77; 12; 9 | — |
| SECONDARY Weight-adjusted Consumption of BAX 855: Average Prophylactic Dose |
45.514; 221.568; 2597.079 | — |
| SECONDARY Weight-adjusted Consumption of BAX 855: Average Number of Prophylactic Infusions |
4.900; 57.067 | — |
| SECONDARY Weight-adjusted Consumption of BAX 855: Average Dose |
55.445; 63.864; 123.510; 417.750; 537.038; 1816.427 | — |
| SECONDARY Number of Participants by Hemostatic Efficacy Rating in Case of Surgery |
10; 0; 0; 1; 11; 0 | — |
| SECONDARY Blood Loss Per Participant in Case of Surgery |
4.8 | — |
| SECONDARY Incremental Recovery (IR) of BAX 855 |
1.546; 1.594; 1.452; 1.651; 1.345; 1.451 | — |
| SECONDARY Half-life (T1/2) of BAX 855 |
— | — |
| SECONDARY Immune Tolerance Induction (ITI) - Number of Participants With Partial Success and Failure of ITI |
1; 1 | — |
| SECONDARY Immune Tolerance Induction (ITI) - Number of Participants With At Least One Catheter-related Complication |
0; 2 | — |
| SECONDARY Immune Tolerance Induction (ITI) - Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies |
0; 1; 0; 0; 0; 1 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Participant is 200 cells per cubic millimeter (mm^3), as confirmed by the central laboratory at screening.
- Parent or legally authorized representative is willing and able to comply with the requirements of the protocol.
Additional inclusion criteria for Part B (immune tolerance induction [ITI]).
- Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion.
- 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
- poorly controlled bleeding despite increased BAX 855 doses, or
- requires bypassing agents to treat bleeding.
Exclusion Criteria
- Participant has detectable FVIII inhibitory antibodies (>=0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
- Participant has a history of FVIII inhibitory antibodies (>=0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening.
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
- Participant has been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for >=3 EDs at any time prior to screening.
- Participant receives > two EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion.
- The participant's weight is anticipated to be 5 times upper limit of normal alanine aminotransferase [ALT], aspartate aminotransferase [AST], or a documented international normalized ratio [INR] >1.5) in his medical history or at the time of screening.
- Participant has severe renal impairment (serum creatinine >1.5 times the upper limit of normal).
- Participant has current or recent ( =0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory.
- Inability or unwillingness to comply with the protocol.
Data sourced from ClinicalTrials.gov (NCT02615691). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.