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Clinical Trial Summary

This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A (intrinsic factor VIII [FVIII] level <1%) without FVIII inhibitors. The study is designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at 3 milligrams per kilogram of body weight (mg/kg) once every 2 weeks (Q2W) for 52 weeks. After 1 year of treatment, participants will continue to receive emicizumab (1.5 mg/kg once every week [QW], 3 mg/kg Q2W or 6 mg/kg once every 4 weeks [Q4W]) over a 7-year long-term follow-up period under this study frame.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04431726
Study type Interventional
Source Hoffmann-La Roche
Contact
Status Active, not recruiting
Phase Phase 3
Start date February 4, 2021
Completion date May 20, 2030

See also
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