NCT02618915

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

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	DTX101	solution for IV infusion

Timeline

• Start date: 2015-12-16
• Primary completion: 2017-10-18
• Completion: 2017-10-18
• First posted: 2015-12-02
• Last updated: 2018-11-14
• Results posted: 2018-11-14

Locations

9 sites across 3 countries: United States, Bulgaria, United Kingdom

Regulatory

• FDA-regulated drug study