Clinical Trial: Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults Wit
Brief Summary: A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.
Detailed Summary:
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.
Study 101HEMB02 is a long-term follow-up study to evaluate the safety, tolerability, and efficacy of AAVrh10-mediated gene therapy of human FIX in subjects with moderate/severe to severe hemophilia B. The primary objective of the study is to determine the long-term safety and efficacy of DTX101 following a single IV infusion (administered during Study 101HEMB01) in adults with moderate/severe to severe hemophilia B.
Sponsor: Dimension Therapeutics
Current Primary Outcome:
- Incidence of adverse events and serious adverse events by dosing group [ Time Frame: 208 weeks ]
- Change from baseline in FIX activity level [ Time Frame: 208 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Number of bleeding episodes requiring recombinant FIX infusion [ Time Frame: 208 weeks ]
Original Secondary Outcome: Same as current
Information By: Dimension Therapeutics
Dates:
Date Received: November 21, 2016
Date Started: January 2017
Date Completion: February 2022
Last Updated: January 16, 2017
Last Verified: January 2017