NCT03315455

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 85 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This multicenter, open-label, Phase 3 study with randomized and non-randomized arms is designed to investigate the efficacy, safety, and pharmacokinetics of emicizumab in participants with hemophilia A regardless of factor VIII (FVIII) inhibitor status. Participants greater than or equal to (≥)12 years old 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: Arm A: Emicizumab prophylaxis at 3 milligrams per kilogram (mg/kg) once every week (QW) subcutaneously (SC) for 4 weeks, followed by 1.5 mg/kg QW SC; Arm B: Emicizumab prophylaxis at 3 mg/kg QW SC for 4 weeks, followed by 6 mg/kg once every 4 weeks (Q4W) SC; and Arm C: No prophylaxis (control arm). In addition, pediatric participants less than (\<)12 years old with hemophilia A and FVIII inhibitors who received episodic therapy with bypassing agents prior to study entry will be enrolled to Arm D: Emicizumab prophylaxis at 3 mg/kg QW SC for 4 weeks, followed by 1.5 mg/kg QW SC.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Emicizumab	Emicizumab will be administered via subcutaneous (SC) injection, as described for each treatment arm.

Timeline

• Start date: 2018-04-26
• Primary completion: 2022-08-03
• Completion: 2025-08-29
• First posted: 2017-10-20
• Last updated: 2026-04-01
• Results posted: 2024-03-06

Locations

13 sites across 4 countries: China, Hong Kong, Malaysia, Thailand