Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

Overview

The primary objective of this study is to assess the percentage of time patients were in normal glucose control.

Full Title of Study: “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)”

Study Type

• Study Type: Observational
• Study Design
  • Time Perspective: Retrospective
• Study Primary Completion Date: February 2021

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

• Other: No Intervention
  • A retrospective chart review of medical records

Clinical Trial Outcome Measures

Primary Measures

• 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 Measures

• Total number of low glycemic events (< 70 mg/dL) over a seven-day period
  • Time Frame: 7 days
• Percentage of time where low glucose values were (< 70mg/dL) over a seven-day period
  • Time Frame: 7 days

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 8 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

• Lead Sponsor
  • Ultragenyx Pharmaceutical Inc
• Provider of Information About this Clinical Study
  • Sponsor
• Overall Official(s)
  • Medical Director, Study Director, Ultragenyx Pharmaceutical Inc