valoctocogene roxaparvovec
Sponsors
BioMarin Pharmaceutical
Conditions
Blood DisorderClotting DisordersGene TherapyHemophilia AHemophilia A With Anti Factor VIIIHemophilia A With InhibitorSevere Haemophilia A
Phase 1
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
CompletedNCT02576795
Start: 2015-09-28End: 2024-02-14Updated: 2025-04-10
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
TerminatedNCT03520712
Start: 2018-04-24End: 2024-08-07Updated: 2025-08-22
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
CompletedNCT04684940
Start: 2020-12-10End: 2026-04-20Updated: 2026-07-10
Phase 3
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
CompletedNCT03370913
Start: 2017-12-19End: 2024-11-20Updated: 2025-03-25
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
CompletedNCT03392974
Start: 2018-03-14End: 2023-06-05Updated: 2023-10-03
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
CompletedNCT04323098
Start: 2020-12-08End: 2025-05-08Updated: 2025-10-08
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
Active, not recruitingNCT06224907
Start: 2023-12-25End: 2029-03-01Target: 6Updated: 2026-03-19
Related Papers
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