Status:
TERMINATED
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Glycogen Storage Disease Type III
Eligibility:
All Genders
1+ years
Brief Summary
The primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric participants with glycogen storage disease type III (GSD III).
Eligibility Criteria
Inclusion
- Key
- Diagnosis of GSD III, confirmed by amylo-alpha-1,6-glucosidase 4-alpha-glucanotransferase (AGL) sequencing or glycogen debranching enzyme (GDE) enzymatic testing
- Key
Exclusion
- Participant is unwilling to remain blinded to continuous glucose monitor (CGM) data for the first 13 weeks of the study, or the Investigator determines that blinding would compromise subject safety
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation or pose undue risk
- Use of any IP or investigational medical device within 30 days or 5.5 half-lives, whichever is longer, prior to screening, or during the study
- Note: Other criteria may apply per protocol
Key Trial Info
Start Date :
May 20 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
March 2 2023
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT05196165
Start Date
May 20 2022
End Date
March 2 2023
Last Update
September 28 2023
Active Locations (4)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
2
Baylor College of Medicine
Houston, Texas, United States, 77030
3
University of Texas
Houston, Texas, United States, 77030
4
University of Groningen Beatrix Children's Hospital
Groningen, Netherlands, 9700RB