Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
25 participants
INTERVENTIONAL
2017-01-26
2023-12-05
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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SPK-8011 5x10^11 vg/kg
Participants received a single intravenous (IV) infusion of SPK-8011 5x10\^11 vector genomes per kilogram (vg/kg) body weight.
SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene
SPK-8011 1x10^12 vg/kg
Participants received a single IV infusion of SPK-8011 1x10\^12 vg/kg.
SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene
SPK-8011 2x10^12 vg/kg
Participants received a single IV infusion of SPK-8011 2x10\^12 vg/kg.
SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene
SPK-8011 1.5x10^12 vg/kg
Participants received a single IV infusion of SPK-8011 1.5x10\^12 vg/kg.
SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene
Interventions
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SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
18 Years
MALE
No
Sponsors
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Spark Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Director
Role: STUDY_DIRECTOR
Spark Therapeutics, Inc.
Locations
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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
Countries
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References
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George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.
Ran G, Chen X, Xie Y, Zheng Q, Xie J, Yu C, Pittman N, Qi S, Yu FX, Agbandje-McKenna M, Srivastava A, Ling C. Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors. Mol Ther Methods Clin Dev. 2020 Mar 13;17:545-555. doi: 10.1016/j.omtm.2020.03.007. eCollection 2020 Jun 12.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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SPK-8011-101
Identifier Type: -
Identifier Source: org_study_id