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An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia A (Kids ALONG)
The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.
Details
| Lead sponsor | Bioverativ Therapeutics Inc. |
|---|---|
| Phase | Phase 3 |
| Status | COMPLETED |
| Enrolment | 71 |
| Start date | 2012-11 |
| Completion | 2013-12 |
Conditions
- Hemophilia A
Interventions
- BIIB031 (rFVIIIFc)
- FVIII (PK subgroup only)
Primary outcomes
- Occurrence of FVIII Inhibitor Development — Up to Week 26 +/- 7 days, or up to 50 exposure days (EDs) if reached prior to Week 26
An inhibitor test result ≥0.6 Bethesda units (BU)/mL, confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Incidences were summarized for any positive inhibitor for participants with ≥50 EDs to rFVIIIFc. In addition, the incidence for all participants, regardless of their EDs to rFVIIIFc, was also summarized. An exact 95% CI for the proportion of participants with a confirmed inhibitor was calculated using the Clopper-Pearson exact method for a binomial proportion.
Countries
United States, Australia, Hong Kong, Ireland, Netherlands, Poland, South Africa, United Kingdom