Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Stopped early · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Glycogen Storage Disease Type III

In brief

The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).

Key facts

Study ID

NCT04990388

Run by

Ultragenyx Pharmaceutical Inc

People needed

9

Starts

2021-10-18

Expected to finish

2023-03-20

Last updated by the study team

2024-04-16

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of California, Irvine — Orange, California, United States
• Rare Disease Research — Atlanta, Georgia, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Texas, Health Science Center of Houston — Houston, Texas, United States
• Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico — Milan, Italy
• Hospital Universitario 12 de Octubre — Madrid, Spain

Full record on ClinicalTrials.gov

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