A Gene Transfer Study for Hemophilia A

Status: Recruiting
Phase:
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:

Contacts:

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

Know Your Options

Not sure which clinical trials might be right for your child? Email our clinical trials team at clinicaltrials@danafarberbostonchildrens.org.
We can help you navigate your options.

Get Clinical Trial Updates

 

Stay informed about Dana-Farber/Boston Children's research efforts, including information on new and current clinical trials. Sign up to receive our email newsletter Advances in Pediatric Hematology/Oncology.

Download Our Clinical Trials

Download a list of our open clinical trials. Check back regularly for updates.