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Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
Sponsor: Ultragenyx Pharmaceutical Inc
Summary
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
Official title: A Long-Term Follow-up Study to Evaluate Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
11
Start Date
2018-08-30
Completion Date
2029-12
Last Updated
2026-02-19
Healthy Volunteers
Not specified
Interventions
No Intervention
No Intervention
Locations (9)
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