Phase 3
N=108
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT02234323 ↗Enrolled (actual)
108
Serious AEs
58.3%
Results posted
Aug 2020
Primary outcome: Primary: Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay — 31.11 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rFVIIIFc (Biological)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- Bioverativ, a Sanofi company
- Primary completion
- Sep 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay |
31.11 | — |
| SECONDARY Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) |
2.24; 1.49; 0.00 | — |
| SECONDARY Annualized Number of Spontaneous Joint Bleeding Episodes |
0.00; 0.00; 0.00 | — |
| SECONDARY Number of rFVIIIFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale |
102; 16; 2; 118; 163; 27 | — |
| SECONDARY Total Number of Exposure Days (EDs) |
100.0 | — |
| SECONDARY Total Annualized rFVIIIFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes |
197.6; 5384.4; 67310.0 | — |
| SECONDARY Number of Injections of rFVIIIFc Required to Resolve a Bleeding Episode |
1.0; 1.0; 1.0 | — |
| SECONDARY Average Dose Per Injection of rFVIIIFc Required to Resolve a Bleeding Episode |
45.45; 48.08; 189.44 | — |
| SECONDARY Change From Baseline in rFVIIIFc Incremental Recovery (IR) |
-0.5; -0.7; -0.4; -0.5; -0.4; -0.8 | — |
| SECONDARY Number of Participants With Response to Immune Tolerance Induction (ITI) |
5; 2; 3; 5 | — |
Summary
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A. The secondary objectives were to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in PUPs, to evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in participants with inhibitors.
Eligibility Criteria
Key Inclusion Criteria
- Ability of the participant's legally authorized representative (e.g. their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
- Weight >=3.5 kg at the time of screening.
- Severe hemophilia A defined as less than (<) 1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period.
Key Exclusion Criteria
- Any exposure to blood components, factor VIII replacement products, including commercially available rFVIIIFc at any time prior to or during screening.
- History of positive inhibitor testing. A prior history of inhibitors was defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (ie, equal to or above lower level of detection).
- History of hypersensitivity reactions associated with any rFVIIIFc administration.
- Other coagulation disorder(s) in addition to hemophilia A.
- Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment.
- Current systemic treatment with chemotherapy and/or other immunosuppressant drugs.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT02234323). 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.