BioMarin Pharmaceutical: Hemophilia A trials
The registry lists 10 Hemophilia A studies with BioMarin Pharmaceutical as lead sponsor. 1 are open to participants now and 5 have results posted.
Phase 1 3Phase 2 3Phase 3 4No phase 3(a study in two phases counts in both)
Studies
Open to participants first, then studies with results posted, then the rest; newest registration first within each.
- A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)Enrolling by invitationNCT05768386
- Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia ACompletedNCT04323098Phase 3Results posted
- Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5TerminatedNCT03520712Phase 1/2Results posted
- Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kgCompletedNCT03392974Phase 3Results posted
- Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)CompletedNCT03370913Phase 3Results posted
- Gene Therapy Study in Severe Haemophilia A Patients (270-201)CompletedNCT02576795Phase 1/2Results posted
- Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia AActive, not recruitingNCT06224907Phase 3
- A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)TerminatedNCT05580692Not applicable
- Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior InhibitorsCompletedNCT04684940Phase 1/2
- A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia ACompletedNCT04560933