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

Glycogen Storage Disease Type IA Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04708015
• Other study ID #: DTX401-CL001
• Status: Completed
• Start date: November 5, 2020
• Est. completion date: March 4, 2022

Study information

• Verified date: May 2022
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: March 4, 2022
• Est. primary completion date: March 4, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type I
• Glycogen Storage Disease Type IA

Intervention

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

Locations

Country	Name	City	State
Netherlands	University Medical Center Groningen	Groningen
United States	UT Health - McGovern Medical School	Houston	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Total number of low glycemic events (< 70 mg/dL) over a seven-day period		7 days
Secondary	Percentage of time where low glucose values were (< 70mg/dL) over a seven-day period		7 days