Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Plain-language summary

This study follows the long-term outcomes of adults with late-onset OTC (ornithine transcarbamylase) deficiency who received the gene therapy DTX301. It includes patients who completed the Week 52 visit of the earlier 301OTC01 study. It is a non-phase follow-up study that is active, not recruiting.

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

Active, not recruiting

Sponsor

Ultragenyx Pharmaceutical Inc

Intervention

No Intervention

Start date

8/30/2018

Estimated completion

12/1/2029

Countries

United States, Canada, France, Spain, United Kingdom

Related genes

Locations

• Aurora, United States
• Boston, United States
• New York, United States
• Cleveland, United States
• Calgary, Canada
• Bron, France
• Santiago de Compostela, Spain
• Barakaldo, Spain
• Birmingham, 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. Completed the Week 52 visit in Study 301OTC01.
2. Willing and able to provide written informed consent.
3. 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 DTX301 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.