Clinical Trial: A Gene Transfer Study for Hemophilia A

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Gene Transfer, Dose-Finding Safety, Tolerability, and Efficacy Study of SPK-8011 [a Recombinant Adeno-Associated Viral Vector With Human Factor VIII Gene] in Individuals With Hemophilia A

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.

Detailed Summary:

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.


Sponsor: Spark Therapeutics

Current Primary Outcome:

  • Number of study-related adverse events, including clinically significant abnormal laboratory values [ Time Frame: 52 weeks ]
  • Changes from baseline in FVIII activity levels after a single outpatient administration of SPK-8011 [ Time Frame: 52 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Kinetic assessment of SPK-8011 including shedding of vector DNA in bodily fluids [ Time Frame: 52 weeks ]
  • Number of participants requiring a course of steroid therapy for the elevations in liver enzymes [ Time Frame: 52 weeks ]


Original Secondary Outcome: Same as current

Information By: Spark Therapeutics

Dates:
Date Received: December 16, 2016
Date Started: December 2016
Date Completion: December 2019
Last Updated: April 27, 2017
Last Verified: April 2017