Clinical Trial: Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief Summary: 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.

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.

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.


Sponsor: Dimension Therapeutics

Current Primary Outcome:

  • The incidence of treatment-related adverse events by dosing group [ Time Frame: 52 weeks ]
  • Change from baseline in FIX activity level [ Time Frame: 6 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome: Number of Bleeding Episodes requiring recombinant FIX infusion [ Time Frame: 52 weeks ]

Original Secondary Outcome: Same as current

Information By: Dimension Therapeutics

Dates:
Date Received: November 23, 2015
Date Started: November 2015
Date Completion: September 2017
Last Updated: May 10, 2017
Last Verified: May 2017