NCT04684940

A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

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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Interventions

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

Study Locations

FacilityCityStateCountry
Children's Hospital Los AngelesLos AngelesCaliforniaUnited States
Hemocentro Da UNICAMPCampinasBrazil
Arthur De Siqueira Cavalcanti Hematology State InstituteRio de JaneiroBrazil
Chaim Sheba Medical CenterRamat GanIsrael
Kyung Hee University Hospital at GangdongSeoulSouth Korea
Kaohsiung Medical University - Chung-Ho Memorial HospitalKaohsiung CityTaiwan
Taichung Veterans General HospitalTaichungTaiwan
National Taiwan University HospitalTaipeiTaiwan
Ege University School of MedicineIzmirTurkey (Türkiye)

Official Trial Information

View on ClinicalTrials.gov

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