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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

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

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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

Most Recent Events

  • 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).

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