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

Running, not enrolling

Conditions studied: Ornithine Transcarbamylase (OTC) Deficiency

In brief

Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.

Key facts

Study ID: NCT03636438
Run by: Ultragenyx Pharmaceutical Inc
People needed: 11
Starts: 2018-08-30
Expected to finish: 2029-12-01
Last updated by the study team: 2026-06-01

Who can join

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

You may qualify if…

Completed the Week 52 visit in Study 301OTC01.
Willing and able to provide written informed consent.
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 DTX301 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

The Children's Hospital Colorado — Aurora, Colorado, United States
Boston Children's Hospital — Boston, Massachusetts, United States
Icahn School of Medicine — New York, New York, United States
University Hospital Cleveland Medical Center/Case Western Reserve University — Cleveland, Ohio, United States
M.A.G.I.C. Clinic — Calgary, Alberta, Canada
Hopital Femme Mere Enfant — Bron, Rhone, France
Hospital Clinico Universitario de Santiago — Santiago de Compostela, Coruna, Spain
Hospital Universitario de Cruces. Servicio de Pediatria — Barakaldo, Vizcaya, Spain
Queen Elizabeth Hospital, Department of Endocrinology — Birmingham, United Kingdom

Full record on ClinicalTrials.gov

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