Phase 3
N=33
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT02234310 ↗Enrolled (actual)
33
Serious AEs
69.7%
Results posted
Jul 2020
Primary outcome: Primary: Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay — 3.03 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rFIXFc (Biological)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- Bioverativ, a Sanofi company
- Primary completion
- Aug 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay |
3.03 | — |
| SECONDARY Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) |
0.21; 1.24 | — |
| SECONDARY Annualized Number of Spontaneous Joint Bleeding Episodes |
0.00; 0.00 | — |
| SECONDARY Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale |
22; 0; 0; 58; 50; 6 | — |
| SECONDARY Total Number of Exposure Days (EDs) |
76.0 | — |
| SECONDARY Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes |
203.2; 3175.0 | — |
| SECONDARY Number of Injections of rFIXFc Required to Resolve a Bleeding Episode |
1.0; 1.0 | — |
| SECONDARY Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode |
88.50; 71.92 | — |
| SECONDARY Change From Baseline in rFIXFc Incremental Recovery (IR) |
0.0; 0.0; -0.0; -0.0; -0.1; -0.1 | — |
Summary
The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs.
Eligibility Criteria
Key Inclusion Criteria
- Weight >=3.5 kilogram at the time of informed consent.
- Severe hemophilia B was defined as less than or equal to ( )200 particles/mcL, or any other known congenital or acquired immunodeficiency).
- Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes was allowed. Treatment in these circumstances should not have exceeded a 14-day duration.
- Participation within the past 30 days in any other clinical study involving investigational treatment.
- Current enrollment in any other clinical study involving investigational treatment.
- Inability to comply with study requirements.
- Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, would have made the participant unsuitable for enrollment.
NOTE: Other protocol-defined inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT02234310). 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.