Hemophilia A-Gene Transfer - Healing Genes

Hemophilia A-Gene Transfer

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A Gene Transfer Study for Hemophilia A

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

Phase 1 / 2

Doctors at three research hospitals seek male patients with Hemophilia A (with Factor VIII Inhibitors) to trial a new product, SPK-8011, an investigatory gene therapy. This therapy utilizes a harmless virus to deliver a functional copy of the Factor VIII (F8) gene which may provide sufficient blood coagulation and reduce need for transfusions.

The treatment requires a single outpatient intravenous injection of SPK-8016 and follow up will involve lab assessments and check-ins over the following year.


  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Not have received gene therapy treatment in the prior year
  • Be HIV- negative


  1. Screening before the treatment
  2. A single infusion of the gene therapy drug will be administered.
  3. Monitoring of the patient for a few hours after dosing, with periodic lab assessments required for 52 weeks.

Trials will take place at 9 locations. Find the location nearest to you.
Contact: Clinical Director  |  215-220-9300  |  [email protected]
Spark Therapeutics
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