Status:
ACTIVE_NOT_RECRUITING
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Hemophilia A With Inhibitor
Hemophilia A With Anti Factor VIII
Eligibility:
MALE
18+ years
Phase:
PHASE1
PHASE2
Brief 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 subje...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
December 10 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2029
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04684940
Start Date
December 10 2020
End Date
April 1 2029
Last Update
July 14 2025
Active Locations (9)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Hemocentro Da UNICAMP
Campinas, Brazil
3
Arthur De Siqueira Cavalcanti Hematology State Institute
Rio de Janeiro, Brazil
4
Chaim Sheba Medical Center
Ramat Gan, Israel