Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

NCT04198987 | Completed

• 1 other identifier: 18-007276
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

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).

Conditions

Congenital Disorder of Glycosylation

Outcome Measures

Primary Outcomes (4)

• Measuring effects of oral monosaccharide supplements through growth measurements

  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.

  length of study, up to 2 years

• Measuring effects of oral monosaccharide supplements through blood sugar levels

  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.

  length of study, up to 2 years

• Measuring effects of oral monosaccharide supplements through liver function results

  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.

  length of study, up to 2 years

• Measuring effects of oral monosaccharide supplements through coagulation results

  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.

  length of study, up to 2 years

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

We will enroll patients diagnosed with congenital disorders of glycosylation and started on oral simple sugar supplements as part of their routine clinical care.

You may qualify if:

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

You may not qualify if:

• Aldolase B deficiency
• Galactosemia
• Hemolytic uremic syndrome
• Severe anemia
• Galactose intolerance

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Congenital Disorders of Glycosylation

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• David Deyle, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: December 8, 2019
• First Posted: December 13, 2019
• Study Start: December 6, 2018
• Primary Completion: June 11, 2025
• Study Completion: June 11, 2025
• Last Updated: October 1, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: length of study

Locations

US (1)