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NCT04323098

A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients

Study Summary

This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.

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Interventions

valoctocogene roxaparvovecBIOLOGICAL
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Study Locations

FacilityCityStateCountry
University of California Davis HealthSacramentoCaliforniaUnited States
Washington University School of MedicineSt LouisMissouriUnited States
Nationwide Children's HospitalColumbusOhioUnited States
The Royal Adelaide HospitalAdelaideAustralia
Royal Brisbane and Women's HospitalBrisbaneAustralia
Alfred HospitalMelbourneAustralia
Fiona Stanley HospitalPerthAustralia
Royal Prince Alfred HospitalSydneyAustralia
Campinas University Clinical HospitalCampinasBrazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São PauloSão PauloBrazil
Changhua Christian Medical Foundation Changhua Christian HospitalChanghuaTaiwan
Taichung Veterans General HospitalTaichungTaiwan

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026