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Phase 3 open-label single-arm study of BMN 270 gene therapy in adults with severe hemophilia A on prophylactic factor VIII
MOH_2018-12-06_002223Phase 3Recruiting
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Dec 2017
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of valoctocogene roxaparvovec in hemophilia A, sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: Safety of BMN 270. Started Dec 2017; status: recruiting.
02
Drugs
valoctocogene roxaparvovec
03
Conditions
hemophilia A
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