Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

Ultragenyx

Status: Terminated

Conditions: Glycogen Storage Disease Type III

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT05196165

UX053-CL002

Details and patient eligibility

About

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).

Enrollment: 14 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• 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 Criteria:

• 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