clinical trial · NCT03636438
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) DeficiencyNo Intervention
brief summary
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
started
Aug 30, 2018
primary completion
Dec 1, 2029
completion
Dec 1, 2029
last updated
Feb 19, 2026
detailed description
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.
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol