drugset / Trial / NCT02234310
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2014-11-13
- Primary completion
- 2019-08-20
- Completion
- 2019-08-20
Publications
- Nolan B, Recht M, Rendo P, Falk A, Foster M, Casiano S, Rauch A, Shapiro A. Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B-LONG study. Eur J Haematol. 2024 Oct;113(4):485-492. doi: 10.1111/ejh.14252. Epub 2024 Jun 25.
- Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Adv. 2021 Jul 13;5(13):2732-2739. doi: 10.1182/bloodadvances.2020004085.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | EFTRENONACOG ALFA | Protein / enzyme biologic | 50 iu/kg | Intravenous |