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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05196165
Recruitment Status : Terminated (Sponsor decision not related to safety concerns)
First Posted : January 19, 2022
Last Update Posted : March 8, 2023
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Tracking Information
First Submitted Date December 10, 2021
First Posted Date January 19, 2022
Last Update Posted Date March 8, 2023
Actual Study Start Date May 20, 2022
Actual Primary Completion Date March 2, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 4, 2022)		 Number of Hypoglycemic Events During the 26-week Observation Period [ Time Frame: Week 26 ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: May 26, 2022)
  • Infant Toddler Quality of Life Questionnaire 47-item Short Form (ITQOL-SF47) Scores [ Time Frame: Up to Week 26 ]
  • Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Fatigue [ Time Frame: Up to Week 26 ]
  • Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Physical Function in Adults [ Time Frame: Up to Week 26 ]
  • 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 ]
  • Short Form 10 (SF10) Domain Scores [ Time Frame: Up to Week 26 ]
  • Short Form 10 (SF10) Component Scores for Children [ Time Frame: Up to Week 26 ]
  • Short Form 36 (SF-36) Component Scores for Adults [ Time Frame: Up to Week 26 ]
  • Short Form 36 (SF-36) Domain Scores [ Time Frame: Up to Week 26 ]
Original Secondary Outcome Measures
 (submitted: January 4, 2022)
  • 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 ]
  • Infant Toddler Quality of Life Questionnaire 47-item Short Form (ITQOL-SF47) Scores [ Time Frame: Up to Week 26 ]
  • Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Fatigue [ Time Frame: Up to Week 26 ]
  • Patient-reported Outcomes Measurement Information System (PROMIS) Questionnaires Scores of the Following Domain: Physical Function in Adults [ Time Frame: Up to Week 26 ]
  • 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 ]
  • Short Form 10 (SF10) Domain Scores [ Time Frame: Up to Week 26 ]
  • Short Form 10 (SF10) Component Scores for Children [ Time Frame: Up to Week 26 ]
  • Short Form 36 (SF-36) Component Scores for Adults [ Time Frame: Up to Week 26 ]
  • Short Form 36 (SF-36) Domain Scores [ Time Frame: Up to Week 26 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
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
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).
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Glycogen Storage Disease Type III
Intervention Other: No Intervention
No investigational product (IP) will be administered in this study
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Terminated
Actual Enrollment
 (submitted: October 31, 2022)		 14
Original Estimated Enrollment
 (submitted: January 4, 2022)		 25
Actual Study Completion Date March 2, 2023
Actual Primary Completion Date March 2, 2023   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages 1 Year and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Netherlands,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT05196165
Other Study ID Numbers UX053-CL002
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Current Responsible Party Ultragenyx Pharmaceutical Inc
Original Responsible Party Same as current
Current Study Sponsor Ultragenyx Pharmaceutical Inc
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators
Study Director: Medical Director Ultragenyx Pharmaceutical Inc
PRS Account Ultragenyx Pharmaceutical Inc
Verification Date March 2023