Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ? 1 IU/dL and Pre-existing Antibodies Against AAV5

This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

Recruitment

Summary

Participation Requirements

Description

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