Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
Summary
The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy by comparing the annualized number of bleeding episodes between participants receiving rFIXFc on each prevention (prophylaxis) regimen and participants receiving rFIXFc on an episodic regimen. The secondary objectives of the study were: to evaluate and assess the pharmacokinetic (PK) parameter estimates of rFIXFc and rFIX (BeneFIX®) at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 (±1 week); to evaluate participants' response to treatment; to evaluate rFIXFc consumption.
Timeline
- Start
- 2009-12
- Primary completion
- 2012-07
- Completion
- 2012-07
Outcome
Met primary endpoint
registry Arm 1: Weekly Prophylaxis vs Arm 3: Episodic (On Demand); p <0.001; Bleeding Rate Ratio 0.17 (95% CI 0.11 to 0.24); negative binomial model NCT01027364 ↗
registry Arm 2: Individualized Interval Prophylaxis vs Arm 3: Episodic (On Demand); p <0.001; Bleeding Rate Ratio 0.13 (95% CI 0.08 to 0.20); negative binomial model NCT01027364 ↗
Publications
- Results Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4.
- Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | EFTRENONACOG ALFA | Protein / enzyme biologic | 20 iu/kg | Intravenous |
| Subject | EFTRENONACOG ALFA | Protein / enzyme biologic | 50 iu/kg | Intravenous |
| Subject | EFTRENONACOG ALFA | Protein / enzyme biologic | 100 iu/kg | Intravenous |
| Subject | rFIX | Protein / enzyme biologic | 50 iu/kg | Intravenous |