A Retrospective Observational Clinical Study on the Use of Continuous Glucose Monitoring for Glycemic Control in Adult and Pediatric Patients With Glycogen Storage Disease Type Ia (GSDIa)
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Glycogen Storage Disease Type IA
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Enrollment
- 15
- Locations
- 2
- Primary Endpoint
- Percentage of time spent in normal glucose control (defined as glucose levels between 70 mg/dL - 120 mg/dL) over a seven-day period
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
The primary objective of this study is to assess the percentage of time patients were in normal glucose control.
Detailed Description
Retrospective Dexcom G6 iCGM data stored in the Clarity cloud from eligible patients will be accessed and downloaded, stripped of patient-identifying information and provided to the contract research organization (CRO) to review prior to sending to the Sponsor for analysis of glycemic control. The CRO will ensure data shared with the Sponsor is de-identified and transferred over a secure file transfer platform. Further, Remote Site Specialists (RSSs) from the CRO will work with the healthcare providers (HCPs) from participating sites to obtain additional patient data including medical history, prescribed diet, cornstarch/Glycosade use and demographic data directly, if available, from the medical charts and will enter this information into an electronic data capture (EDC) system.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of GSDIa confirmed by either liver biopsy or molecular testing.
- •Currently using the Dexcom G6 iCGM for glycemic control and with approximately one (1) month of CGM data available on the Dexcom Clarity cloud and have authorized sharing of data with thereferral center through the Dexcom Clarity app.
Exclusion Criteria
- •Presence or history of any disease, condition or chronic medication that, in the Investigator's opinion, would significantly affect interpretation of study results.
- •Use of any investigational product or investigational medical device within 30 calendar days preceding screening or before completion of all scheduled study assessments during study enrollment.
Outcomes
Primary Outcomes
Percentage of time spent in normal glucose control (defined as glucose levels between 70 mg/dL - 120 mg/dL) over a seven-day period
Time Frame: 7 days
Secondary Outcomes
- Percentage of time where low glucose values were (< 70mg/dL) over a seven-day period(7 days)
- Total number of low glycemic events (< 70 mg/dL) over a seven-day period(7 days)