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This study examines SB-525 (PF-07055480), a gene therapy for people with severe hemophilia A that aims to deliver the gene for the missing clotting factor into cells. It was open to men aged 18 and over with severe hemophilia A. It was conducted in 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: * Male ≥18 years of age * Severe hemophilia A (past evidence of circulating FVIII activity of \< 1% normal) * Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days * ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months * Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion Exclusion Criteria: * Presence of neutralizing antibodies * Current inhibitor, or history of FVIII inhibitor (except for transient low titer inhibitor detected in childhood) * History of hypersensitivity response to FVIII * History of Hepatitis B or HIV-1/2 infection * History of Hepatitis C, unless viral assays in two samples, collected at least 6 months apart, are negative * Evidence of any bleeding disorder in addition to hemophilia A * Markers of hepatic inflammation or overt or occult cirrhosis * History of chronic renal disease or creatinine ≥ 1.5 mg/dL * Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha fetoprotein * Presence of \> grade 2 liver fibrosis on elastography for subjects with history of treated Hepatitis C or suspicion of chronic liver disease