Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Completed · Phase 1/Phase 2
Conditions studied: Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
In brief
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.
Key facts
- Study ID
- NCT04684940
- Run by
- BioMarin Pharmaceutical
- People needed
- 10
- Starts
- 2020-12-10
- Expected to finish
- 2026-04-20
- Last updated by the study team
- 2026-07-10
Who can join
Age: 18 and older. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- Males ≥ 18 years of age with hemophilia A and documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent.
- History of a positive inhibitor result with the first positive result at least 12 month prior to Screening.
- Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer < 0.6 BU.
- Prophylactic or on-demand hemophilia therapy in the last 12 months. Bleeding, inhibitor \& hemophilia therapy Hx over previous 12 months.
- Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion.
- Willing to abstain from consumption of alcohol for at least the first 52 weeks following BMN 270 infusion.
You may not qualify if…
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded.
- Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only).
- Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
- Evidence of any bleeding disorder not related to hemophilia A.
Where it is running
- Children's Hospital Los Angeles — Los Angeles, California, United States
- Hemocentro Da UNICAMP — Campinas, 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)
- Royal Free Hospital — London, United Kingdom
Full record on ClinicalTrials.gov
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