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

Hemophilia B Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02971969
• Other study ID #: 101HEMB02
• Secondary ID: 2016-003430-25
• Status: Completed
• Start date: January 2017
• Est. completion date: November 6, 2021

Study information

• Verified date: January 2022
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: November 6, 2021
• Est. primary completion date: November 6, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Locations

Country	Name	City	State
United Kingdom	Haemophilia, Haemostasis & Thrombosis Centre	Basingstoke	Hampshire
United Kingdom	Manchester Haemophilia Comprehensive Care Center	Manchester
United States	University of Michigan Hospital and Health Systems	Ann Arbor	Michigan
United States	UF CRC - Clinical Research Center	Gainesville	Florida
United States	Arkansas Children's Hospital	Little Rock	Arkansas

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events and serious adverse events by dosing group		208 weeks
Primary	Change from baseline in FIX activity level		208 weeks
Secondary	Number of bleeding episodes requiring recombinant FIX infusion		208 weeks