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

Terminated

• Conditions: Glycogen Storage Disease Type III

• Registration Number: NCT05196165
• Lead Sponsor: Ultragenyx Pharmaceutical Inc

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-12-10
• Study First Submitted QC: 2022-01-04
• Study First Posted: 2022-01-19
• Study Start: 2022-05-20
• Primary Completion: 2023-03-02
• Study Completion: 2023-03-02
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-26
• Last Update Posted: 2023-09-28

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 14

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Hypoglycemic Events During the 26-week Observation Period	Week 26

Secondary Outcome Measures

Name	Time	Method
Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores	Up to Week 26
Number of Times the Participant can Rise from a Seated to a Standing Position in a 30-second Period, as Measured by the Site-to-Stand (STS) Test	Up to Week 26
Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores	Up to Week 26
Ankle Dorsiflexion, Measured Bilaterally Using Goniometry	Up to Week 26
Percent Predicted Distance Walked as Measured by 6-minute Walk Test (6MWT) and 12-minute Walk Test (12MWT)	Up to Week 26
GNE Myopathy Functional Activities Scale (GNEM-FAS) Expanded Version Domain Scores and Total Score	Up to Week 26
Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)	Up to Week 26

Trial Locations

Locations (4)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

University of Texas; 🇺🇸 Houston, Texas, United States

University of Groningen Beatrix Children's Hospital; 🇳🇱 Groningen, Netherlands