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
Phase of Trial: Phase I/II
Latest Information Update: 07 Mar 2018
At a glance
- Drugs DTX 301 (Primary)
- Indications Inborn urea cycle disorders
- Focus Adverse reactions; First in man
- Acronyms CAPtivate
- Sponsors Dimension Therapeutics
- 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.
- 07 Mar 2018 Results presented in the Ultragenyx Pharmaceutical Media Release.
- 07 Mar 2018 According to an Ultragenyx Pharmaceuticals media release, As of the February 15, 2018 data cutoff date, patient 1 has been followed for 24 weeks, patient 2 for 20 weeks, and patient three for 12 weeks.
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Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History