Project Title

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

Official Title

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.

Project 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.

Blood Disorder

  • Hemophilia A
  • Severe Hemophilia A

Patient Recruitment Details

Patient recruitment status: Closed

Number of Patients (globally)

245 participants.

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