Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04198987

Enrollment

Registered

2019-12-13

Start date

2018-12-06

Completion date

2025-06-11

Last updated

2025-10-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital Disorder of Glycosylation

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

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

Design outcomes

Primary

Measure	Time frame	Description
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 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.
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.

Countries

United States

Outcome results