Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Completed · Phase 1/Phase 2
Conditions studied: GSD1
In brief
The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
Key facts
- Study ID
- NCT03517085
- Run by
- Ultragenyx Pharmaceutical Inc
- People needed
- 12
- Starts
- 2018-05-18
- Expected to finish
- 2021-11-02
- Last updated by the study team
- 2022-11-18
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCONN Health — Farmington, Connecticut, United States
- Michigan Medicine University of Michigan — Ann Arbor, Michigan, United States
- UT Health - McGovern Medical School — Houston, Texas, United States
- Montreal Children Hospital, McGill University Health Centre — Montreal, Quebec, Canada
- University Medical Center Groningen — Groningen, Netherlands
- Complejo Hospitalario Universitario de Santiago — Santiago de Compostela, A Coruna, Spain
Full record on ClinicalTrials.gov
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