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

Plain-language summary

This is a long-term follow-up study evaluating the safety, tolerability and efficacy of the gene therapy DTX101 (AAVrh10FIX) in adult males with moderate/severe to severe hemophilia B. To take part, participants must have completed the Week 52 visit of the preceding Study 101HEMB01. Participants are also expected to be willing to stop prophylactic recombinant FIX treatment at specified time points during the study when medically acceptable.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

Ultragenyx Pharmaceutical Inc

Start date

1/1/2017

Estimated completion

11/6/2021

Countries

United States, United Kingdom

Related genes

Locations

• Little Rock, United States
• Gainesville, United States
• Ann Arbor, United States
• Basingstoke, United Kingdom
• Manchester, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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