Brief Title
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
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 With Moderate/Severe to Severe Hemophilia B
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 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. 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.
Study Type
Observational
Primary Outcome
Incidence of adverse events and serious adverse events by dosing group
Secondary Outcome
Number of bleeding episodes requiring recombinant FIX infusion
Condition
Hemophilia B
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
6
Start Date
January 2017
Completion Date
February 18, 2022
Primary Completion Date
February 18, 2022
Eligibility Criteria
Inclusion Criteria: 1. Willing and able to provide written informed consent. 2. Completed the Week 52 visit in Study 101HEMB01. 3. Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable. 4. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements. Exclusion Criteria: 1. 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. 2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Medical Director, ,
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT02971969
Organization ID
101HEMB02
Responsible Party
Sponsor
Study Sponsor
Ultragenyx Pharmaceutical Inc
Study Sponsor
Medical Director, Study Director, Ultragenyx Pharmaceutical Inc
Verification Date
March 2021