Gene Transfer Clinical Study in Crigler-Najjar Syndrome
Stopped early · Phase 1
Conditions studied: Crigler-Najjar Syndrome
In brief
This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year. Subjects will receive a single dose of AT342 and will be followed for safety and efficacy for 5 years.
Key facts
- Study ID
- NCT03223194
- Run by
- Audentes Therapeutics
- People needed
- 1
- Starts
- 2017-09-08
- Expected to finish
- 2021-02-11
- Last updated by the study team
- 2022-05-18
Who can join
Age: 1 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Children's Hospital at Montefiore — The Bronx, New York, United States
- Clinic for Special Children — Strasburg, Pennsylvania, United States
- Shaare Zedek Medical Center — Jerusalem, Israel
- King's College Hospital NHS Foundation Trust — London, United Kingdom
Full record on ClinicalTrials.gov
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