Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: OTC Deficiency
In brief
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
Key facts
- Study ID
- NCT05345171
- Run by
- Ultragenyx Pharmaceutical Inc
- People needed
- 37
- Starts
- 2022-10-18
- Expected to finish
- 2031-03-01
- Last updated by the study team
- 2026-06-02
Who can join
Age: 12 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California — Los Angeles, California, United States
- University of Colorado — Aurora, Colorado, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
- Hospital Italiano de Buenos Aires — Buenos Aires, Argentina
- Clinica Universitaria Reina Fabiola — Córdoba, Argentina
- Hospital de Clinicas de Porto Alegre — Porto Alegre, Brazil
- The Hospital for Sick Children — Toronto, Ontario, Canada
- Hopital Femme Mere Enfant — Bron, France
- Necker-Enfants Maladas Hospital — Paris, France
- Universitatsklinikum Heidelberg — Heidelberg, Germany
- Kumamoto University Hospital — Kumamoto, Japan
- Fujita Health University Hospital — Toyoake, Japan
- Erasmus Universitair Medisch Centrum Rotterrdam — Rotterdam, Netherlands
- Centro Hospitalar Universitario de Sao Joao — Porto, Portugal
- Fundacio Hospital Universitari Vall D'Hebron-Institute de Recerca — Barcelona, Spain
Full record on ClinicalTrials.gov
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