Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

Recruiting

• Conditions: Congenital Disorder of Glycosylation

• Registration Number: NCT04198987
• Lead Sponsor: Mayo Clinic

Brief Summary

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).

Detailed Description

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.

Study Timeline

• Study Start: 2018-12-06
• Study First Submitted: 2019-12-08
• Study First Submitted QC: 2019-12-12
• Study First Posted: 2019-12-13
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-14
• Last Update Posted: 2025-01-15
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Measuring effects of oral monosaccharide supplements through growth measurements	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	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	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	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.

Trial Locations

Locations (1)

Mayo Clinic in Rochester; 🇺🇸 Rochester, Minnesota, United States