Study 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.
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Valoctocogene RoxaparvovecBIOLOGICAL
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center | Johannesburg | South Africa | |
| Kyung Hee University Hospital at Gangdong | Seoul | South Korea | |
| Severance Hospital, Yonsei University Health System | Seoul | South Korea | |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | Taiwan | |
| Taichung Veterans General Hospital | Taichung | Taiwan | |
| National Taiwan University Hospital | Taipei | Taiwan | |
| Tri-Service General Hospital | Taipei | Taiwan | |
| Royal Free Hospital | London | United Kingdom | |
| University Hospital Southampton NHS Foundation Trust | Southampton | United Kingdom |