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A Gene Transfer Study for Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03003533
Recruitment Status : Active, not recruiting
First Posted : December 28, 2016
Last Update Posted : March 30, 2023
Information provided by (Responsible Party):
Spark Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Hemophilia A Genetic: SPK-8011 Phase 1 Phase 2

Detailed Description:

Hemophilia A is a condition in which blood is unable to clot effectively. It is caused by a mutation or deletion in the gene that is responsible for producing blood-clotting factor VIII protein. Individuals with hemophilia A suffer from repeated bleeding episodes, often into the joints, which can cause chronic joint disease and sometime results in death due to the inability of the blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden. The current treatment is intravenous (i.v.) injections of factor VIII protein products, either 2-3 times weekly or in response to bleeding.

Recent preliminary clinical data of a hemophilia B gene transfer study (which is also being conducted by Spark Therapeutics) shows all study participants achieving therapeutic factor IX activity levels (average of maintaining factor IX activity levels around 30% of normal with no confirmed bleeds, after receiving Spark gene transfer, with the approach of using the novel bio-engineered recombinant adeno-associated viral (rAAV) vector carrying a high specific activity of a factor IX gene. The approach being tested in this clinical research study uses a further modified novel AAV vector (with a stronger attraction to the human liver) to deliver the human factor VIII (hFVIII) gene into liver cells so that they can produce factor VIII protein.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : January 26, 2017
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: SPK-8011
All participants who meet the eligibility criteria will receive an outpatient single intravenous (i.v.) administration of SPK-8011.
Genetic: SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene

Primary Outcome Measures :
  1. Number of study-related adverse events, including clinically significant abnormal laboratory values [ Time Frame: 52 weeks ]
    adverse events

  2. Changes from baseline in FVIII activity levels after a single outpatient administration of SPK-8011 [ Time Frame: 52 weeks ]
    changes in FVIII activity levels

Secondary Outcome Measures :
  1. Kinetic assessment of SPK-8011 including shedding of vector DNA in bodily fluids [ Time Frame: 52 weeks ]
    vector shedding

  2. Number of participants requiring a course of steroid therapy for the elevations in liver enzymes [ Time Frame: 52 weeks ]
    number of participants requiring steroids

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03003533

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United States, California
University of California Davis - Hemostasis and Thrombosis Center
Sacramento, California, United States, 94817
United States, Florida
University of Florida Health
Gainesville, Florida, United States, 32610
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Mississippi
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States, 39110
United States, New York
Weill Cornell Medicine-Comprehensive Center for Hemophilia and Coagulation Disorders
New York, New York, United States, 10065
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Pennsylvania State University Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 10733
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Jefferson University Hospitals
Philadelphia, Pennsylvania, United States, 19107
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
United States, Virginia
Virginia Commonwealth University School of Medicine
Richmond, Virginia, United States, 23219
Australia, New South Wales
Royal Prince Alfred Hosptial
Camperdown, New South Wales, Australia, 2050
The Alfred Hospital
Melbourne, Australia, 3004
Canada, Ontario
McMaster University Medical Centre and Juravinski Hospital
Hamilton, Ontario, Canada, L8N 3Z5
Chaim Sheba Center
Ramat Gan, Tel Hashomer, Israel, 526000
Mahidol University - Ramathibody Hospital
Bangkok, Thailand, 10400
Sponsors and Collaborators
Spark Therapeutics
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Study Director: Clinical Trial Director Spark Therapeutics, Inc.
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Spark Therapeutics Identifier: NCT03003533    
Other Study ID Numbers: SPK-8011-101
First Posted: December 28, 2016    Key Record Dates
Last Update Posted: March 30, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Spark Therapeutics:
Adeno-Associated Virus (AAV)
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Factor VIII (FVIII) Deficiency
Factor VIII (FVIII) Gene
Factor VIII (FVIII) Protein
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Gene Therapy
Gene Transfer
Hematologic Diseases
Hemorrhagic Disorders
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn