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This study examines SPK-8011, a gene transfer therapy, in men with hemophilia A (a bleeding disorder caused by a lack of clotting factor FVIII). The therapy aims to deliver a gene that enables the body to produce the missing clotting factor. It enrolled men aged 18 and over with low FVIII levels. The study was carried out in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Males age 18 years or older * Confirmed diagnosis of hemophilia A as evidenced by their medical history with baseline FVIII activity levels \<=2% * Have received \>150 exposure days (EDs) to FVIII concentrates or cryoprecipitate * Have no prior history of allergic reaction to any FVIII product * Have no measurable inhibitor against FVIII as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein and no clinical signs or symptoms of decreased response to FVIII administration * Agree to use reliable barrier contraception Exclusion Criteria: * Evidence of active hepatitis B or C * Currently on antiviral therapy for hepatitis B or C * Have significant underlying liver disease * Have serological evidence\* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 and who are on an antiretroviral drug regimen (\* participants who are HIV+ and stable with CD4 count \>200/mm3 and undetectable viral load are eligible to enroll) * Have detectable antibodies reactive with AAV-Spark200 capsid * Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks