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

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

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.

Study Overview

• Status: Completed
• Conditions: 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
• Enrollment (Actual): 6

Contacts and Locations

Study Locations

• United Kingdom
  • Manchester, United Kingdom, M13 9WL
    • Manchester Haemophilia Comprehensive Care Center
  • Hampshire
    • Basingstoke, Hampshire, United Kingdom, RG24 9NA
      • Haemophilia, Haemostasis & Thrombosis Centre
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • Florida
    • Gainesville, Florida, United States, 32610
      • UF CRC - Clinical Research Center
  • Michigan
    • Ann Arbor, Michigan, United States, 48109-5872
      • University of Michigan Hospital and Health Systems

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01 clinical study

Description

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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events and serious adverse events by dosing group	208 weeks
Change from baseline in FIX activity level	208 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of bleeding episodes requiring recombinant FIX infusion	208 weeks

Collaborators and Investigators

• Sponsor: Ultragenyx Pharmaceutical Inc

Study record dates

Study Major Dates

• Study Start: January 1, 2017
• Primary Completion (Actual): November 6, 2021
• Study Completion (Actual): November 6, 2021

Study Registration Dates

• First Submitted: November 21, 2016
• First Submitted That Met QC Criteria: November 21, 2016
• First Posted (Estimate): November 23, 2016

Study Record Updates

• Last Update Posted (Actual): January 6, 2022
• Last Update Submitted That Met QC Criteria: January 4, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Keywords: Hemophilia B; gene therapy
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: 101HEMB02; 2016-003430-25 (EudraCT Number)