Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

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

Start Date

January 2017

Completion Date

November 6, 2021

Primary Completion Date

November 6, 2021

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

Secondary IDs

2016-003430-25

Responsible Party

Sponsor

Study Sponsor

Ultragenyx Pharmaceutical Inc

Study Sponsor

Medical Director, Study Director, Ultragenyx Pharmaceutical Inc

Verification Date

January 2022