Hemophilia B Clinical Trial
Official title:
Phase I/II Open-Label Safety and Dose Finding Study of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the
factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation.
Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to
chronic arthropathy (joint damage), intracranial hemorrhage, and even death. In patients with
moderate/severe to severe hemophilia B, the majority of bleeding episodes occur in the joints
and, if not treated, lead to debilitating damage and a decreased quality of life.
This study will evaluate the safety and efficacy of the adeno-associated virus (AAV) to
deliver human factor IX (hFIX) gene, the healthy gene necessary to make FIX, to the liver
where FIX is normally produced. This study will determine if AAVrh10 can produce clinically
meaningful FIX levels in patients with moderately/severe or severe hemophilia B.
This study was previously posted by Dimension Therapeutics, which has been acquired by
Ultragenyx in November 2017.
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