Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants (HAVEN 5)

This randomized, multicenter, open-label, Phase 3 study is designed to investigate the efficacy, safety, and pharmacokinetics of emicizumab in participants with hemophilia A regardless of factor VIII (FVIII) inhibitor status. Participants who received episodic therapy with FVIII or bypassing agents prior to study entry and experienced at least 5 bleeds over the prior 24 weeks will be randomized in a 2:2:1 ratio to the following regimens: 1) Emicizumab prophylaxis at 3 milligrams per kilogram (mg/kg) every week (QW) subcutaneously (SC) for 4 weeks, followed by 1.5 mg/kg QW SC; 2) Emicizumab prophylaxis at 3 mg/kg QW SC for 4 weeks, followed by 6 mg/kg every 4 weeks (Q4W) SC; 3) No prophylaxis (control arm).


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Patients
Anticipated Study Start Date: April 28, 2018
Estimated Primary Completion Date: August 28, 2019
Estimated Study Completion Date: February 18, 2020

Arms and Interventions:
- Experimental: Prophylactic Emicizumab 1.5 mg/kg QW
- Experimental: Prophylactic Emicizumab 6 mg/kg Q4W
- Control Arm: No Prophylaxis