NCT02502149

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale

A Randomized, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) Manufactured at 15K Scale and at Different Vial Strengths in Previously Treated Subjects With Severe Hemophilia A

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Bioverativ Therapeutics Inc. · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to compare the pharmacokinetic (PK) of recombinant coagulation factor VIII Fc fusion protein (rFVIIIFc) manufactured at the current scale of 2000 L (2K) to the PK of rFVIIIFc manufactured at the 15,000 L (15K) scale in previously treated participants with severe hemophilia A. The secondary objectives are: to characterize the PK of rFVIIIFc manufactured at the 15K scale at the 15K baseline and after 13 weeks of treatment; to characterize the PK of rFVIIIFc manufactured at the 15K scale at 1000 IU/vial and 6000 IU/vial strengths; and to evaluate the safety of rFVIIIFc manufactured at the 15K scale.

Detailed description

PK assessments are in 3 phases: Pharmacokinetic Assessment 1(PK1): PK assessments following single injection of rFVIIIFc manufactured at the 2K scale. Pharmacokinetic Assessment 2 (PK2): PK assessments are made following a single injection of rFVIIIFc manufactured at the 15K scale where participants are randomized to the 1000 IU vial or 6000 IU/vial strengths. Pharmacokinetic Assessment 3 (PK3): PK assessments are made following 13 weeks of rFVIIIFc treatment manufactured at the 15K scale where participants are randomized to the 1000 IU vial or 6000 IU/vial strengths. After study completion, in countries where rFVIIIFc is not commercially available, eligible participants will be offered enrollment into a long-term safety and efficacy extension study (8HA01EXT \[NCT01454739\]).

Conditions

• Severe Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	rFVIIIFc	As per arm description

Timeline

• Start date: 2015-08-01
• Primary completion: 2017-04-01
• Completion: 2017-06-01
• First posted: 2015-07-20
• Last updated: 2020-12-19
• Results posted: 2018-04-06

Locations

15 sites across 3 countries: United States, Australia, New Zealand