Glycogen Storage Disease Type IA
Conditions
Keywords
Dexcom G6 iCGM, glucose metabolism disorder, CGM, Continuous Glucose Monitoring
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.
Interventions
OTHERNo Intervention
A retrospective chart review of medical records
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Observational model
CASE_ONLY
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
8 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of time spent in normal glucose control (defined as glucose levels between 70 mg/dL - 120 mg/dL) over a seven-day period | 7 days |
Secondary
| Measure | Time frame |
|---|---|
| Total number of low glycemic events (< 70 mg/dL) over a seven-day period | 7 days |
| Percentage of time where low glucose values were (< 70mg/dL) over a seven-day period | 7 days |
Countries
Netherlands, United States
Outcome results
None listed