Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
Study 301OTC02 is a long-term follow-up study to evaluate the safety and efficacy of adeno-associated virus (AAV) serotype 8 (AAV8)-mediated gene transfer of human OTC in adults with late-onset OTC deficiency. Only subjects who complete Study 301OTC01 (NCT02991144) are eligible to participate in Study 301OTC02.
Study Type
OBSERVATIONAL
Enrollment
11
No Intervention
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
Number of Participants with Adverse Events and Serious Adverse Events
Time frame: Up to 416 weeks
Change from Baseline Over Time in the Ureagenesis Rate
Sodium acetate is used as a tracer to measure the rate of ureagenesis
Time frame: Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration
Change from Baseline Over Time in 24-Hour Area Under the Curve for Plasma Ammonia
Time frame: Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration
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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