Glycogen Storage Disease Type IANCT04708015
Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
An observational study of Glycogen Storage Disease Type IA, run by Ultragenyx Pharmaceutical Inc at 2 sites in 2 countries.
Trial summary
The primary objective of this study is to assess the percentage of time patients were in normal glucose control. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Glycogen Storage Disease Type IA
- How many people
- 15 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 5 Nov 2020to 4 Mar 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
UT Health - McGovern Medical School
Houston, Texas, 77030
About this study
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)
The primary objective of this study is to assess the percentage of time patients were in normal glucose control.
Read the 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.
- Conditions
- Glycogen Storage Disease Type IA
- Being tested
- No Intervention
- Keywords
- Dexcom G6 iCGM, glucose metabolism disorder, CGM, Continuous Glucose Monitoring
- Medical subject headings
- Hepatorenal form of glycogen storage disease, Glucose Metabolism Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
A retrospective chart review of medical records
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of time spent in normal glucose control (defined as glucose levels between 70 mg/dL - 120 mg/dL) over a seven-day period
Measured: 7 days
Secondary outcomes (2)
Total number of low glycemic events (< 70 mg/dL) over a seven-day period
Measured: 7 days
Percentage of time where low glucose values were (< 70mg/dL) over a seven-day period
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2021
- Study start5 Nov 2020
- Primary completion4 Mar 2022
- Study completion4 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IAStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04708015
- Sponsor's protocol ID
- DTX401-CL001
- Status last verified
- May 2024
- First submitted
- 11 Jan 2021
- Last updated
- 29 May 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04708015
- Acronym / sponsor ID
- DTX401-CL001
- Conditions
- Glycogen Storage Disease Type IA
- Keywords
- Dexcom G6 iCGM, glucose metabolism disorder, CGM, Continuous Glucose Monitoring
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 5 Nov 2020 (actual)
- Primary completion
- 4 Mar 2022 (actual)
- Study completion
- 4 Mar 2022 (actual)
- First posted
- 13 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionA retrospective chart review of medical records | Other | – |
Primary outcome
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
2 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Ultragenyx Pharmaceutical Inc
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type IA, run by Ultragenyx Pharmaceutical Inc at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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 (time frame: 7 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2024.