Hemophilia | Overview
Hemophilia A is a genetic bleeding disorder caused by a mutation in the F8 gene that leads to low levels of a protein called factor VIII. Factor VIII is necessary for blood clotting. Severe hemophilia A can cause prolonged or spontaneous bleeding, particularly in the muscles and joints, or internal organs. The current standard of care for hemophilia A is to control or prevent bleeding episodes through factor replacement therapy, given by regular intravenous (IV) infusions.
Hemophilia A gene therapy clinical trial
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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 |
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Recruiting males 18 Years and older (full eligibility criteria) |
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Boston Children’s Hospital |
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Started: Dec. 2016 |
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Stacy Croteau, MD, principal investigator |
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Colleen Dansereau, MSN, RN, CPN: 617-919-7008 or colleen.dansereau@childrens.harvard.edu |
