This study investigated a gene therapy called SPK-9001 in patients with hemophilia B, an inherited bleeding disorder. It enrolled men over 18 with a confirmed diagnosis who experienced an average of at least 4 bleeding events per year. It was a Phase 2 study 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: * Able to provide informed consent and comply with requirements of the study * Males ≥18 y.o. with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous factor IX) * Received ≥50 exposure days to factor IX products * A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions * No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein * Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences 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 (\* subjects who are HIV+ and stable with CD4 count \>200/mm3 and undetectable viral load are eligible to enroll) * Neutralizing antibodies reactive with AAV-Spark100 above and/or below a defined titre * Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 12 weeks * Unable or unwilling to comply with study assessments