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Sponsor decision not related to safety concerns
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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).
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | No investigational product (IP) will be administered in this study |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Hypoglycemic Events During the 26-week Observation Period | Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores | Up to Week 26 | |
| Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores | Up to Week 26 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other criteria may apply per protocol
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States | ||
| Baylor College of Medicine |
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| Label | URL |
|---|---|
| Patient Advocacy at Ultragenyx: Glycogen Storage Disease Type III (GSDIII) | View source |
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| 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 |
| Ankle Dorsiflexion, Measured Bilaterally Using Goniometry | 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 |
| Houston |
| Texas |
| 77030 |
| United States |
| University of Texas | Houston | Texas | 77030 | United States |
| University of Groningen Beatrix Children's Hospital | Groningen | 9700RB | Netherlands |
| ID | Term |
|---|---|
| D006010 | Glycogen Storage Disease Type III |
| ID | Term |
|---|---|
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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