A Gene Transfer Study for Hemophilia A

Status: Recruiting
DFCI Protocol ID:

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.

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Stacy Croteau, MD, Children's Hospital Boston

Site-responsible Investigators:


Children's Hospital Boston: Colleen Dansereau, Colleen.dansereau@childrens.harvard.edu

Eligibility Criteria

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 inhibitor 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

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