Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

Completed

Conditions studied: Glycogen Storage Disease Type III

In brief

The primary objective of this study is to evaluate potential biomarkers of GSD III.

Key facts

Study ID

NCT04574830

Run by

Ultragenyx Pharmaceutical Inc

People needed

18

Starts

2020-11-23

Expected to finish

2022-06-30

Last updated by the study team

2022-09-13

Who can join

Age: 5 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed historical diagnosis of GSD III based on pathogenic mutations in the AGL gene on both alleles or GDE deficiency based on biopsy of liver, muscle, or fibroblasts
• Willing to comply with all study procedures
• Willing and able to provide written informed consent. If a minor, willing and able to provide written assent and have a legally authorized representative willing and able to provide written informed consent

You may not qualify if…

• Presence or history of any condition that, in the view of the Investigator, places the subject at high risk of poor study compliance, interferes with study participation, or interferes with the subject's ability to safely or reliably complete the study assessments
• Use of any IP within 30 days prior to informed consent/assent or at the time of enrollment

Where it is running

• University of California — Irvine, California, United States
• Children's Hospital Orange County — Orange, California, United States
• Colorado Children's Hospital — Aurora, Colorado, United States
• University of Texas Medical School — Houston, Texas, United States

Full record on ClinicalTrials.gov

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