Trial Profile
A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
Status:
Completed
Phase of Trial:
Phase I/II
Latest Information Update: 30 Jan 2023
Price :
$35
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At a glance
- Drugs Avalotcagene ontaparvovec (Primary) ; Prednisolone; Prednisone
- Indications Ornithine carbamoyltransferase deficiency disease
- Focus Adverse reactions; First in man
- Acronyms CAPtivate
- Sponsors Dimension Therapeutics; Ultragenyx Pharmaceutical
- 03 Jan 2022 Status changed from active, no longer recruiting to completed.
- 29 Nov 2021 Results presented in an Ultragenyx Pharmaceutical media release.
- 29 Nov 2021 According to Ultragenyx Pharmaceutical media release, new long-term data from this study were presented at the 14th International Congress of Inborn Errors of Metabolism (ICIEM).