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

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

Status

Terminated

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05196165

Enrollment

Registered

2022-01-19

Start date

2022-05-20

Completion date

2023-03-02

Last updated

2023-09-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen Storage Disease Type III

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).

Interventions

OTHERNo Intervention

No investigational product (IP) will be administered in this study

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

1 Years to No maximum

Healthy volunteers

Inclusion 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

Design outcomes

Primary

Measure	Time frame
Number of Hypoglycemic Events During the 26-week Observation Period	Week 26

Secondary

Measure	Time frame
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
Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)	Up to Week 26
Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores	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
Ankle Dorsiflexion, Measured Bilaterally Using Goniometry	Up to Week 26

Countries

Netherlands, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026