Gene-transfer, Open-label, Dose-escalation Study of SPK-8011 [Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene] in Individuals With Hemophilia A

SPK-8011-101

This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.

2017-01-26

2023-12-05

2023-12-05

Completed

Early phase I

25

Inclusion Criteria: * Males age18 years or older * Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels ≤ 2% of normal * Have received \>150 exposure days (EDs) to FVIII concentrates or cryoprecipitate * Have experienced \>10 bleeding events over the previous 12 months only if receiving on-demand therapy and having FVIII baseline level 1-2% of normal * Have no prior history of allergic reaction to any FVIII product * Have no measurable inhibitor against Factor VIII as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein * 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 (\* 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 25, 'type': 'ACTUAL'}}

2024-01-09