NCT02172950

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects With Severe Hemophilia A

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 246 (actual)
• Sponsor: CSL Behring · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs) with severe congenital hemophilia A and previous exposure to FVIII products who achieve at least 100 exposure days (EDs) to rVIII-SingleChain in this study, as well as in previously untreated patients (PUPs) with no previous exposure to any FVIII product who achieve at least 50 EDs to rVIII-SingleChain in this study. A substudy (open to both PTPs and PUPs) will investigate the use of rVIII-SingleChain in surgery. A substudy (open to PUPs who develop an inhibitor to rVIII-SingleChain) will investigate the use of rVIII-SingleChain in immune tolerance induction (ITI) therapy.

Conditions

• Hemophilia A
• Severe Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	rVIII-SingleChain	Recombinant single-chain coagulation factor VIII

Timeline

• Start date: 2014-10-13
• Primary completion: 2021-01-19
• Completion: 2021-01-19
• First posted: 2014-06-24
• Last updated: 2021-10-27
• Results posted: 2021-10-27

Locations

64 sites across 25 countries: United States, Australia, Austria, Canada, Czechia, France, Georgia, Germany, Hungary, Ireland, Italy, Japan, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Romania, South Africa, Spain, Switzerland, Thailand, Ukraine, United Kingdom