Phase 3
N=240
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT01454739 ↗Enrolled (actual)
240
Serious AEs
25.0%
Results posted
Nov 2018
Primary outcome: Primary: Number of Participants With Any Positive Inhibitor Development — 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rFVIIIFc (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- Male
- Sponsor
- Bioverativ Therapeutics Inc.
- Primary completion
- Oct 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Any Positive Inhibitor Development |
0; 0; 0 | — |
| SECONDARY Annualized Bleeding Rate (ABR) |
1.18; 1.59; 0.64; 1.90; 3.72; 1.01 | — |
| SECONDARY Annualized Spontaneous Joint Bleeding Episodes |
0.00; 0.00; 0.00; 0.58; 2.20; 0.00 | — |
| SECONDARY Total Number of Exposure Days (EDs) |
287.00; 373.00; 257.00; 203.50; 127.00; 107.0 | — |
| SECONDARY Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg]) |
5417.9; 4989.7; 4359.8; 3505.2; 5457.1; 4572.3 | — |
| SECONDARY Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale |
403; 1061; 24; 186; 1; 5 | — |
| SECONDARY Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale |
139; 155; 475; 260; 9; 1 | — |
Summary
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
Eligibility Criteria
Key Inclusion Criteria
- Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
- Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).
Key Exclusion Criteria
- Confirmed positive high-titer inhibitor (≥5.00 BU/mL).
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01454739). 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.