Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec With Prophylactic Steroids in Hemophilia A

  • End date
    Sep 24, 2026
  • participants needed
  • sponsor
    BioMarin Pharmaceutical
Updated on 24 July 2021


This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.

Condition Hemophilia, hema, Hemophilia A
Treatment valoctocogene roxaparvovec
Clinical Study IdentifierNCT04323098
SponsorBioMarin Pharmaceutical
Last Modified on24 July 2021


Yes No Not Sure

Inclusion Criteria

Males 18 years of age with hemophilia A and residual FVIII levels 1 IU/dL as evidenced by medical history, at the time of signing the informed consent
Must have been on prophylactic hemophilia therapy for at least 12 months prior to study entry
Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs)
No previous documented history of a detectable FVIII inhibitor, <0.6 Bethesda Units (BU)
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

Exclusion Criteria

Detectable pre-existing antibodies to the AAV5 capsid. Up to 25% of subjects may have detectable pre-existing AAV5 capsid antibodies, so long as the titer level is below the minimum required dilution (< 20)
Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded
Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy
Evidence of any bleeding disorder not related to hemophilia A
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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