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

Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05196165
Collaborator
(none)
25
Enrollment
4
Locations
8.4
Anticipated Duration (Months)
6.3
Patients Per Site
0.7
Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease Intervention/Treatment Phase
  • Other: No Intervention

Study Design

Study Type:
Observational
Anticipated Enrollment :
25 participants
Observational Model:
Case-Only
Time Perspective:
Prospective
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
Actual Study Start Date :
May 20, 2022
Anticipated Primary Completion Date :
Feb 1, 2023
Anticipated Study Completion Date :
Feb 1, 2023

Outcome Measures

Primary Outcome Measures

  1. Number of Hypoglycemic Events During the 26-week Observation Period [Week 26]

Secondary Outcome Measures

  1. Infant Toddler Quality of Life Questionnaire 47-item Short Form (ITQOL-SF47) Scores [Up to Week 26]

  2. Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Fatigue [Up to Week 26]

  3. Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Physical Function in Adults [Up to Week 26]

  4. Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Physical Activity in 5-17 Year Olds [Up to Week 26]

  5. Short Form 10 (SF10) Domain Scores [Up to Week 26]

  6. Short Form 10 (SF10) Component Scores for Children [Up to Week 26]

  7. Short Form 36 (SF-36) Component Scores for Adults [Up to Week 26]

  8. Short Form 36 (SF-36) Domain Scores [Up to Week 26]

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
2 Baylor College of Medicine Houston Texas United States 77030
3 University of Texas Houston Texas United States 77030
4 University of Groningen Beatrix Children's Hospital Groningen Netherlands 9700RB

Sponsors and Collaborators

  • Ultragenyx Pharmaceutical Inc

Investigators

  • Study Director: Medical Director, Ultragenyx Pharmaceutical Inc

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier:
NCT05196165
Other Study ID Numbers:
  • UX053-CL002
First Posted:
Jan 19, 2022
Last Update Posted:
Aug 24, 2022
Last Verified:
Aug 1, 2022
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Glycogen Storage Disease
Glycogen Storage Disease Type III
Hypoglycemia
Disease

Study Results

No Results Posted as of Aug 24, 2022