Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

Researchers are trying to assess whether the use of simple sugars given as a daily dietary supplement can improve the health of children with congenital disorders of glycosylation (CDG).

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Key information

Conditions

Congenital Disorder of Glycosylation Carbohydrate Metabolism, Inborn Errors Congenital Disorders of Glycosylation Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

About this study

The goal of this study is to collect data from patients diagnosed with congenital disorders of glycosylation and taking a simple sugar supplement. The study team wants to expand the evidence on the beneficial effects of this treatment in clinical practice.

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patient has a biochemically and genetically proven congenital disorder of glycosylation
Patient is receiving (or planning to receive) oral simple sugar supplementation

Exclusion criteria

Aldolase B deficiency
Galactosemia
Hemolytic uremic syndrome
Severe anemia
Galactose intolerance

Treatment and study plan

Primary outcomes

Measuring effects of oral monosaccharide supplements through growth measurements

Time frame: length of study, up to 2 years

To assess the effects of oral monosaccharide supplementation for each participant, changes in participant growth parameters, as well as blood sugar levels, coagulation parameters, liver function, and other measures of organ system function (as appropriate for the specific type of CDG) will be correlated with biomarkers derived from participant blood and urine samples obtained at key time points and then compared to standard normative ranges of data for each measure.
Measuring effects of oral monosaccharide supplements through blood sugar levels

Time frame: length of study, up to 2 years

To assess the effects of oral monosaccharide supplementation for each participant, changes in participant growth parameters, as well as blood sugar levels, coagulation parameters, liver function, and other measures of organ system function (as appropriate for the specific type of CDG) will be correlated with biomarkers derived from participant blood and urine samples obtained at key time points and then compared to standard normative ranges of data for each measure.
Measuring effects of oral monosaccharide supplements through liver function results

Time frame: length of study, up to 2 years

To assess the effects of oral monosaccharide supplementation for each participant, changes in participant growth parameters, as well as blood sugar levels, coagulation parameters, liver function, and other measures of organ system function (as appropriate for the specific type of CDG) will be correlated with biomarkers derived from participant blood and urine samples obtained at key time points and then compared to standard normative ranges of data for each measure.
Measuring effects of oral monosaccharide supplements through coagulation results

Time frame: length of study, up to 2 years

To assess the effects of oral monosaccharide supplementation for each participant, changes in participant growth parameters, as well as blood sugar levels, coagulation parameters, liver function, and other measures of organ system function (as appropriate for the specific type of CDG) will be correlated with biomarkers derived from participant blood and urine samples obtained at key time points and then compared to standard normative ranges of data for each measure.

Sponsors and collaborators

Lead sponsor

Mayo Clinic

Other

Registry information

Important dates

Study start: 2018
Primary completion: 2025
Study completion: 2025
First posted: Dec 13, 2019
Registry last updated: Oct 1, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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