MedTrace Logo

Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia

NCT04311307 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2022-02-11

No results posted yet for this study

Summary

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

Conditions

  • Glycogen Storage Disease Type IA

Interventions

BIOLOGICAL

[6,6-2H2]glucose

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.

Sponsors & Collaborators

Study Design

Allocation
NON_RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2021-01-19
Primary Completion
2022-02-07
Completion
2022-02-07

Countries

  • Netherlands

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04311307 on ClinicalTrials.gov