Active — Not RecruitingPhase 1/2

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

United States, Brazil, Israel10 participantsStarted 2020-12-10

Plain-language 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.

Who can participate

Age range18 Years

SexMALE

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Inclusion criteria

✓. 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.
✓. 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 criteria

✕. 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.

What they're measuring

Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.

Timeframe: 60 months

Trial details

NCT IDNCT04684940

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-04

View on ClinicalTrials.gov More Hemophilia A With Inhibitor trials