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

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)

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

Study Overview

• Status: Completed
• Conditions: Glycogen Storage Disease Type IA
• Intervention / Treatment: Other: No Intervention

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.

• Study Type: Observational
• Enrollment (Actual): 16

Contacts and Locations

Study Locations

• Netherlands
  • Groningen, Netherlands, 9700 RB
    • University Medical Center Groningen
• United States
  • Texas
    • Houston, Texas, United States, 77030
      • UT Health - McGovern Medical School

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Male and female patients eight years of age and older with GSDIa, who have used the Dexcom G6 iCGM for glycemic control, and with at least one month of CGM data available on the Dexcom Clarity cloud.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Ultragenyx Pharmaceutical Inc

Study record dates

Study Major Dates

• Study Start (Actual): November 5, 2020
• Primary Completion (Actual): March 4, 2022
• Study Completion (Actual): March 4, 2022

Study Registration Dates

• First Submitted: January 11, 2021
• First Submitted That Met QC Criteria: January 11, 2021
• First Posted (Actual): January 13, 2021

Study Record Updates

• Last Update Posted (Actual): May 10, 2022
• Last Update Submitted That Met QC Criteria: May 9, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: Continuous Glucose Monitoring; CGM; glucose metabolism disorder; Dexcom G6 iCGM
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Disease; Glycogen Storage Disease; Glycogen Storage Disease Type I
• Other Study ID Numbers: DTX401-CL001