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

Completed

• Conditions: Hemophilia B

• Registration Number: NCT02971969
• Lead Sponsor: Ultragenyx Pharmaceutical Inc

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 Timeline

• Study First Submitted: 2016-11-21
• Study First Submitted QC: 2016-11-21
• Study First Posted: 2016-11-23
• Study Start: 2017-01
• Primary Completion: 2021-11-06
• Study Completion: 2021-11-06
• Status Verified: 2022-01
• Last Update Submitted: 2022-01-04
• Last Update Posted: 2022-01-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 6

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change from baseline in FIX activity level	208 weeks
Incidence of adverse events and serious adverse events by dosing group	208 weeks

Secondary Outcome Measures

Name	Time	Method
Number of bleeding episodes requiring recombinant FIX infusion	208 weeks

Trial Locations

Locations (5)

Haemophilia, Haemostasis & Thrombosis Centre; 🇬🇧 Basingstoke, Hampshire, United Kingdom

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

Manchester Haemophilia Comprehensive Care Center; 🇬🇧 Manchester, United Kingdom

UF CRC - Clinical Research Center; 🇺🇸 Gainesville, Florida, United States

University of Michigan Hospital and Health Systems; 🇺🇸 Ann Arbor, Michigan, United States