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

Glycogen Storage Disease Type III Clinical Trial

Official title:

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Patients 1 Year of Age and Over With GSD III

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05196165
• Other study ID #: UX053-CL002
• Status: Terminated
• Start date: May 20, 2022
• Est. completion date: March 2, 2023

Study information

• Verified date: September 2023
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 14
• Est. completion date: March 2, 2023
• Est. primary completion date: March 2, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

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

Related Conditions & MeSH terms

• Disease
• Glycogen Storage Disease
• Glycogen Storage Disease Type III
• Hypoglycemia

Intervention

Other:
• No Intervention
No investigational product (IP) will be administered in this study

Locations

Country	Name	City	State
Netherlands	University of Groningen Beatrix Children's Hospital	Groningen
United States	Baylor College of Medicine	Houston	Texas
United States	University of Texas	Houston	Texas
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Hypoglycemic Events During the 26-week Observation Period		Week 26
Secondary	Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores		Up to Week 26
Secondary	Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores		Up to Week 26
Secondary	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
Secondary	Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)		Up to Week 26
Secondary	Ankle Dorsiflexion, Measured Bilaterally Using Goniometry		Up to Week 26
Secondary	Percent Predicted Distance Walked as Measured by 6-minute Walk Test (6MWT) and 12-minute Walk Test (12MWT)		Up to Week 26
Secondary	GNE Myopathy Functional Activities Scale (GNEM-FAS) Expanded Version Domain Scores and Total Score		Up to Week 26

External Links

•   Patient Advocacy at Ultragenyx: Glycogen Storage Disease Type III (GSDIII)