Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults 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.

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.

This study was previously posted by Dimension Therapeutics, which has been acquired by Ultragenyx.

Inclusion criteria

• Willing and able to provide written informed consent.
• Completed the Week 52 visit in Study 101HEMB01.
• Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable.
• Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.

Exclusion criteria

• Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX101 during the duration of this study.
• Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study