Clinical trial NCT04708015: Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Glycogen Storage Disease Type IA
Keywords: Dexcom G6 iCGM
glucose metabolism disorder
CGM
Continuous Glucose Monitoring
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2021-01-13
Enrollment: 15
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
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.

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.

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.

Primary outcomes:
measure: Percentage of time spent in normal glucose control (defined as glucose levels between 70 mg/dL - 120 mg/dL) over a seven-day period
timeFrame: 7 days
Secondary outcomes:
measure: Total number of low glycemic events (< 70 mg/dL) over a seven-day period
timeFrame: 7 days
measure: Percentage of time where low glucose values were (< 70mg/dL) over a seven-day period
timeFrame: 7 days
Other outcomes:
Locations:
city: Houston
country: United States
facility: UT Health - McGovern Medical School
state: Texas
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
Study officials:
affiliation: Ultragenyx Pharmaceutical Inc
name: Medical Director
role: STUDY_DIRECTOR
References: