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Long-term follow-up after valoctocogene roxaparvovec (Roctavian) gene therapy in severe hemophilia A
2023-507749-27-00Phase 4Completed
- Sponsor
- BioMarin Pharmaceutical
- Start
- Aug 2024
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 14
01
Overview
Phase 4, open-label, single-arm trial of valoctocogene roxaparvovec in hemophilia A, sponsored by BioMarin Pharmaceutical. Primary endpoints: Adverse drug reactions and serious adverse events, Events of special interest – hepatotoxicity and 4 more. Started Aug 2024; status: completed.
02
Drugs
valoctocogene roxaparvovec
03
Conditions
hemophilia A
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