- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04311307
Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia (ENGLUPRO GSDIa)
Endogenous Glucose Production in Subjects With Glycogen Storage Disease Type Ia Estimated by a Single Oral Dose of Stable Isotopes: an Investigator-initiated Human Pilot Study
Glycogen storage disease type Ia (GSDIa) subjects retain a limited capacity for endogenous glucose production (EGP). To date, the origin of residual EGP in GSDIa patients is unknown. Either increased glycogen debranching or lysosomal glycogen breakdown can account for residual EGP in GSDIa.
Innovative treatments for GSDIa (e.g. AAV8-mediated gene therapy and mRNA therapy) are being developed.Therefore, longitudinal minimally-invasive monitoring of outcomes after therapeutic interventions in GSD Ia subjects becomes warranted.
The primary objective is to test the feasibility of EGP quantification in adult GSDIa subjects by stable isotopes after a single oral [6,6-2H2]glucose dose. Secondary objectives are to compare EGP assessed by a single oral [6,6-2H2]glucose dose (a) in GSDIa patients versus matched healthy participants, (b) among GSDIa patients, (c) in the pre-prandial state versus the fed state, (d) in the controlled hospital setting versus the home setting. Data collected from the continuous glucose monitoring data will also be compared
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study design: An investigator-initiated human pilot-study. Study population: Ten adult subjects with GSDIa and ten age and gender-matched healthy subjects.
Interventions: Three experiments will be performed for each subject. During the first in hospital experiment, two oral D-[6,6-2H2]glucose loads will be performed 2 hours before breakfast and at lunchtime, respectively. The third oral D-[6,6-2H2]glucose load will be performed at home (random time). Capillary blood samples will be collected on filter paper at specific time points after each oral D-[6,6-2H2]glucose load. During the experiments, subjects will be monitored by subcutaneous continuous glucose monitoring (CGM).
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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-
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Groningen, Netherlands
- University of Groningen, UMC Groningen
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- The diagnosis should be confirmed by G6PC mutation analysis
- Age above 16 years
- Stable medical condition before the start of the test procedures
Exclusion Criteria:
- Age < 16 years
- Intercurrent illness
- Recent history of hospitalization due to hypoglycemia
- Pregnancy
- History suggestive of diabetes or fasting intolerance
- First grade family member with a confirmed diagnosis associated with fasting intolerance
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Basic Science
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Patients
GSDIa patients
|
Each subject will be administered two oral [6,6-2H2]glucose loads (pre-prandial, fed) in the hospital setting and one oral [6,6-2H2]glucose load at home setting (random time).
Capillary blood samples will be collected on filter paper 10 times at specific time points after each oral [6,6-2H2]glucose load.
|
Active Comparator: Controls
Healthy volunteers
|
Each subject will be administered two oral [6,6-2H2]glucose loads (pre-prandial, fed) in the hospital setting and one oral [6,6-2H2]glucose load at home setting (random time).
Capillary blood samples will be collected on filter paper 10 times at specific time points after each oral [6,6-2H2]glucose load.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
[6,6-2H2]glucose concentration in GSDIa patients
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
Endogenous glucose production (EGP) in GSDIa will be assessed through minimal model calculation
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every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
[6,6-2H2]glucose concentration in GSDIa patients and matched healthy participants
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
[6,6-2H2]glucose concentration in severe and attenuated GSDIa patients
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
[6,6-2H2]glucose concentration in the pre-prandial state versus the fed state in GSDIa patients
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
[6,6-2H2]glucose concentration in the pre-prandial state versus the fed state in healthy participants
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
[6,6-2H2]glucose concentration in the controlled hospital setting versus the home setting in GSDIa patients
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
[6,6-2H2]glucose concentration in the controlled hospital setting versus the home setting in healthy participants
Time Frame: every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
|
Collaborators and Investigators
Collaborators
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- NL73191.042.20
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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