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: 04 Jun 2018

At a glance

  • Drugs DTX 301 (Primary)
  • Indications Ornithine-carbamoyltransferase-deficiency-disease
  • Focus Adverse reactions; First in man
  • Acronyms CAPtivate
  • Sponsors Dimension Therapeutics
  • Most Recent Events

    • 30 May 2018 Planned End Date changed from 1 Nov 2018 to 1 Dec 2019.
    • 30 May 2018 Planned primary completion date changed from 1 Nov 2018 to 1 Dec 2019.
    • 07 Mar 2018 According to an Ultragenyx Pharmaceuticals media release, Data Monitoring Committee (DMC) has completed its review of the current Cohort 1 data. Company will enroll three patients in cohort 2. The first patient is expected to be enrolled in March 2018, and data from the second cohort are expected in the second half of 2018.
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