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
Hemophilia A is an X-linked recessive genetic bleeding disorder caused by mutations in the factor 8 (F8) gene. FVIII is produced in liver endothelial cells and is critical for fibrin clot formation. Severe hemophilia A is characterized by frequent, spontaneous internal bleeding that can lead to chronic joint damage, intracranial hemorrhage, and even death. Current treatment for hemophilia A is based on replacement of the deficient FVIII with IV injections of recombinant FVIII clotting protein prophylactically or as needed to treat bleeding episodes.
This gene therapy clinical trial for hemophilia A evaluates whether the AAV vector carrying a F8 gene can increase FVIII levels in patients with moderate to severe hemophilia A. Specifically, the study will evaluate the safety and efficacy of the adeno-associated virus (AAV) to deliver the human F8 gene, the healthy gene necessary to make FVIII, to the liver. This study will determine if SPK-8011 can produce clinically meaningful FVIII levels in patients with moderately/severe or severe hemophilia A.