Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency — CAPtivate  

Ornithine Transcarbamylase (OTC) Deficiency Clinical Trial

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

Status Active, not recruiting

Clinical Trial Summary

Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Ornithine Transcarbamylase (OTC) Deficiency

• NCT number: NCT03636438
• Study type: Observational
• Source: Ultragenyx Pharmaceutical Inc
• Status: Active, not recruiting
• Start date: August 30, 2018
• Completion date: December 2029