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

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 16 Nov 2017

At a glance

  • Drugs DTX 301 (Primary)
  • Indications Inborn urea cycle disorders
  • Focus Adverse reactions; First in man
  • Acronyms CAPtivate
  • Sponsors Dimension Therapeutics
  • Most Recent Events

    • 16 Nov 2017 According to an Ultragenyx Pharmaceuticals media release, the company has completed patient dosing in the first cohort of three patients enrolled in this trial. The decision to proceed to the next, higher dose cohort will be made after the data monitoring committee evaluates the safety data for all patients in the previous dosing cohort.
    • 31 Aug 2017 According to a Dimension Therapeutics media release, the company has commenced patient dosing in this trial.
    • 27 Jun 2017 According to a Dimension Therapeutics media release, initial data from the study is expected by end of the year 2017.
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