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

Summary

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.

Timeline

Start

2018-04-24

Primary completion

2024-08-07

Completion

2024-08-07

Outcome

Outcome not reported

Stopped: “Early rollover into long-term extension study”

Drugs

Indications