Trial Profile
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, no longer recruiting
Phase of Trial:
Phase I/II
Latest Information Update: 31 Jan 2024
Price :
$35
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At a glance
- Drugs Avalotcagene ontaparvovec (Primary)
- Indications Ornithine carbamoyltransferase deficiency disease
- Focus Adverse reactions
- Acronyms CAPtivate
- Sponsors Ultragenyx Pharmaceutical
- 23 Jan 2024 Planned End Date changed from 1 Dec 2027 to 1 Dec 2029.
- 23 Jan 2024 Planned primary completion date changed from 1 Dec 2027 to 1 Dec 2029.
- 08 May 2023 According to Ultragenyx Pharmaceutical media release, data from this study will be presented at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting