NCT01458106

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 71 (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 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.

Detailed description

Previously treated pediatric participants will be treated with a prophylactic regimen of rFVIIIFc. PK analysis of pre-study factor VIII (FVIII) and rFVIIIFc will be performed in a sub-group of the study participants prior to commencement of prophylactic treatment. After these PK results are available, remaining participants have the option of proceeding directly to prophylactic treatment. After completing the end of study assessments, eligible participants would be able to continue treatment in Study 8HA01EXT.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	BIIB031 (rFVIIIFc)	Vials of rFVIIIFc 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	FVIII (PK subgroup only)	Baseline prestudy FVIII dosing in participants who enter the PK subgroup. Vials of prestudy FVIII 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: 2012-11-01
• Primary completion: 2013-12-01
• Completion: 2013-12-01
• First posted: 2011-10-24
• Last updated: 2020-12-19
• Results posted: 2014-12-12

Locations

23 sites across 8 countries: United States, Australia, Hong Kong, Ireland, Netherlands, Poland, South Africa, United Kingdom