Study Summary
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
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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 |
|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | United States |
| Hemocentro Da UNICAMP | Campinas | Brazil | |
| Arthur De Siqueira Cavalcanti Hematology State Institute | Rio de Janeiro | Brazil | |
| Chaim Sheba Medical Center | Ramat Gan | Israel | |
| Kyung Hee University Hospital at Gangdong | 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 | |
| Ege University School of Medicine | Izmir | Turkey (Türkiye) |