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Valoctocogene Roxaparvovec in Severe Haemophilia A
NCT02576795Phase 2Completed
- Sponsor
- BioMarin Pharmaceutical
- Start
- Sep 2015
- Primary completion
- Feb 2024
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of valoctocogene roxaparvovec in hemophilia A, sponsored by BioMarin Pharmaceutical. Primary endpoints: Treatment Emergent Adverse Events at 7 years, FVIII Activity Levels >= 5 IU/dL at 16 weeks and 1 more. Started Sep 2015; status: completed.
02
Drugs
valoctocogene roxaparvovec
03
Conditions
hemophilia A
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