Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
Completed · Phase 1/Phase 2
Conditions studied: Ornithine Transcarbamylase (OTC) Deficiency
In brief
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.
Key facts
- Study ID
- NCT02991144
- Run by
- Ultragenyx Pharmaceutical Inc
- People needed
- 16
- Starts
- 2017-07-31
- Expected to finish
- 2021-12-16
- Last updated by the study team
- 2023-01-26
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
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 at Mount Sinai — New York, New York, United States
- University Hospital Cleveland Medical Center/Case Western Reserve University — Cleveland, Ohio, United States
- Alberta's Children's Hospital — Calgary, Alberta, Canada
- Hospital Clinico Universitario de Santiago — Santiago de Compostela, A Coruna, Spain
- Hospital Universitario de Cruzes — Barakaldo, Vizcaya, Spain
- National Hospital for Neurology & Neurosurgery — London, London City, United Kingdom
- Queen Elizabeth Hospital — Birmingham, United Kingdom
Full record on ClinicalTrials.gov
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