A Gene Transfer Study for Hemophilia A

Completed · Phase 1/Phase 2

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT03003533

Run by

Spark Therapeutics, Inc.

People needed

25

Starts

2017-01-26

Expected to finish

2023-12-05

Last updated by the study team

2024-12-30

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Males age 18 years or older
• Confirmed diagnosis of hemophilia A as evidenced by their medical history with baseline FVIII activity levels <=2%
• Have received >150 exposure days (EDs) to FVIII concentrates or cryoprecipitate
• Have no prior history of allergic reaction to any FVIII product
• Have no measurable inhibitor against FVIII as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein and no clinical signs or symptoms of decreased response to FVIII administration
• Agree to use reliable barrier contraception

You may not qualify if…

• 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 and who are on an antiretroviral drug regimen (* 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

Where it is running

• University of California Davis - Hemostasis and Thrombosis Center — Sacramento, California, United States
• University of Florida Health — Gainesville, Florida, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Mississippi Center for Advanced Medicine — Madison, Mississippi, United States
• Weill Cornell Medicine-Comprehensive Center for Hemophilia and Coagulation Disorders — New York, New York, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Pennsylvania State University Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Jefferson University Hospitals — Philadelphia, Pennsylvania, United States
• Hemophilia Center of Western Pennsylvania — Pittsburgh, Pennsylvania, United States
• Virginia Commonwealth University School of Medicine — Richmond, Virginia, United States
• Royal Prince Alfred Hosptial — Camperdown, New South Wales, Australia
• The Alfred Hospital — Melbourne, Australia
• McMaster University Medical Centre and Juravinski Hospital — Hamilton, Ontario, Canada
• Chaim Sheba Center — Ramat Gan, Tel Hashomer, Israel
• Mahidol University - Ramathibody Hospital — Bangkok, Thailand

Full record on ClinicalTrials.gov

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