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

N/ATerminatedNCT05196165

Ultragenyx Pharmaceutical Inc14 enrolled

Overview

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).

Study Type

OBSERVATIONAL

Enrollment

Conditions

Glycogen Storage Disease Type III

Interventions

No InterventionOTHER

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

Eligibility

Sex: ALLMin age: 1 Year

Medical Language ↔ Plain English

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

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

Outcomes

Primary Outcomes

Number of Hypoglycemic Events During the 26-week Observation Period

Time frame: Week 26

Secondary Outcomes

Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores

Time frame: Up to Week 26

Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores

Time frame: 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

Time frame: Up to Week 26

Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)

Time frame: Up to Week 26

Ankle Dorsiflexion, Measured Bilaterally Using Goniometry

Time frame: Up to Week 26

Percent Predicted Distance Walked as Measured by 6-minute Walk Test (6MWT) and 12-minute Walk Test (12MWT)

Time frame: Up to Week 26

GNE Myopathy Functional Activities Scale (GNEM-FAS) Expanded Version Domain Scores and Total Score

Time frame: Up to Week 26

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.