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

Completed

Conditions studied: Hemophilia B

In brief

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.

Key facts

Study ID

NCT02971969

Run by

Ultragenyx Pharmaceutical Inc

People needed

6

Starts

2017-01-01

Expected to finish

2021-11-06

Last updated by the study team

2022-01-06

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Willing and able to provide written informed consent.
• Completed the Week 52 visit in Study 101HEMB01.
• Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable.
• Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.

You may not qualify if…

• 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.
• Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study

Where it is running

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

Full record on ClinicalTrials.gov

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