Glycogen Storage Disease III
Conditions
Interventions
None listed
Sponsors
Ultragenyx pharmaceutical Inc.
Eligibility
Age
No minimum to 99 Years
Inclusion criteria
Inclusion criteria: 1. Male or female 1 year of age or older at time of informed consent/assent 2. Diagnosis of GSD III, confirmed by AGL sequencing or GDE enzymatic testing 3. Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures. If a minor or an adult with cognitive limitations, willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related procedures
Exclusion criteria
Exclusion criteria: 1. Subject is unwilling to remain blinded to CGM data, or the Investigator determines that blinding would compromise subject safety 2. Presence or history of any condition that, in the view of the Investigator, would interfere with participation or pose undue risk 3. 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
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Additional details regarding the primary analysis are in the Statistical Analysis Plan (SAP) | — |
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint of this study is the incidence of hypoglycemia during the 26-week Observation Period. | — |
Countries
Netherlands
Outcome results
None listed