NCT01440946

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

An Open-label, Multicenter Evaluation of Safety, Pharmacokinetics and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia B

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Bioverativ Therapeutics Inc. · Industry
• Sex: Male
• Age: 11 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFIXFc; to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes

Detailed description

At the Baseline visit (28 ± 7 days prior to Day 1), participants receive a single IV injection of prestudy FIX over 10 (±5) minutes in the clinic under medical supervision at a dose of 50 IU/kg. A washout period with no FIX treatment is required prior to administration of prestudy FIX and prior to rFIXFc. A PK assessment is done with prestudy FIX and also done with rFIXFc on Day 1. After completing the PK assessments, participants begin weekly prophylactic treatment with rFIXFc for approximately 50 weeks, to obtain 50 EDs. One ED is defined as a 24-hour period in which a participant received 1 or more doses of rFIXFc, with the time of the first injection of rFIXFc defined as the start of the ED.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
DRUG	rFIXFc	Vials of rFIXFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.
DRUG	FIX	Vials of prestudy FIX (provided by the participants) were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.

Timeline

• Start date: 2012-06-01
• Primary completion: 2014-11-01
• Completion: 2014-11-01
• First posted: 2011-09-27
• Last updated: 2020-12-19
• Results posted: 2015-06-18

Locations

15 sites across 6 countries: United States, Australia, Ireland, Netherlands, South Africa, United Kingdom