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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05196165
Recruitment Status : Recruiting
First Posted : January 19, 2022
Last Update Posted : September 26, 2022
Sponsor:
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment
Glycogen Storage Disease Type III Other: No Intervention

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Study Type : Observational
Estimated 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
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : February 2023



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


Primary Outcome Measures :
  1. Number of Hypoglycemic Events During the 26-week Observation Period [ Time Frame: Week 26 ]

Secondary Outcome Measures :
  1. Infant Toddler Quality of Life Questionnaire 47-item Short Form (ITQOL-SF47) Scores [ Time Frame: Up to Week 26 ]
  2. Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Fatigue [ Time Frame: Up to Week 26 ]
  3. Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Physical Function in Adults [ Time Frame: 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 [ Time Frame: Up to Week 26 ]
  5. Short Form 10 (SF10) Domain Scores [ Time Frame: Up to Week 26 ]
  6. Short Form 10 (SF10) Component Scores for Children [ Time Frame: Up to Week 26 ]
  7. Short Form 36 (SF-36) Component Scores for Adults [ Time Frame: Up to Week 26 ]
  8. Short Form 36 (SF-36) Domain Scores [ Time Frame: Up to Week 26 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study aims to enroll at least 5 participants from each of the following age groups: 1-5 years, 6-12 years, 12-17 years, and ≥ 18 years.
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05196165


Contacts
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Contact: Patients Contact: Trial Recruitment 1-888-756-8657 TrialRecruitment@ultragenyx.com
Contact: HCPs Contact: Medical Information 1-888-756-8657 medinfo@ultragenyx.com

Locations
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United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
University of Texas Recruiting
Houston, Texas, United States, 77030
Netherlands
University of Groningen Beatrix Children's Hospital Recruiting
Groningen, Netherlands, 9700RB
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT05196165    
Other Study ID Numbers: UX053-CL002
First Posted: January 19, 2022    Key Record Dates
Last Update Posted: September 26, 2022
Last Verified: September 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glycogen Storage Disease
Glycogen Storage Disease Type III
Hypoglycemia
Disease
Glucose Metabolism Disorders
Metabolic Diseases
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn