Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Patients 1 Year of Age and Over with GSD III.
- Conditions
- Glycogen Storage Disease III10027424
- Registration Number
- NL-OMON51818
- Lead Sponsor
- ltragenyx pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 2
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
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
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint of this study is the incidence of hypoglycemia during the<br /><br>26-week Observation Period. </p><br>
- Secondary Outcome Measures
Name Time Method <p>Additional details regarding the primary analysis are in the Statistical<br /><br>Analysis Plan (SAP)</p><br>