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Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04198987
Recruitment Status : Recruiting
First Posted : December 13, 2019
Last Update Posted : December 17, 2019
Sponsor:
Information provided by (Responsible Party):
Eva Morava-Kozicz, 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).

Condition or disease
Congenital Disorder of Glycosylation

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.

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Study Type : Observational
Estimated Enrollment : 15 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation
Actual Study Start Date : December 6, 2018
Estimated Primary Completion Date : December 1, 2024
Estimated Study Completion Date : December 31, 2024





Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04198987


Locations
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United States, Minnesota
Mayo Clinic in Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Eva Morava-Kozicz, MD, PhD    507-266-2967    morava-kozicz.eva@mayo.edu   
Contact: Suzy Boyer, RDN, LD    507-266-2967    boyer.suzanne@mayo.edu   
Sponsors and Collaborators
Mayo Clinic
Investigators
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Principal Investigator: Eva Morava-Kozicz, MD, PhD Mayo Clinic
  Study Documents (Full-Text)

Documents provided by Eva Morava-Kozicz, Mayo Clinic:
Study Protocol  [PDF] November 19, 2018

Additional Information:
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Responsible Party: Eva Morava-Kozicz, Principal Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT04198987    
Other Study ID Numbers: 18-007276
First Posted: December 13, 2019    Key Record Dates
Last Update Posted: December 17, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Data will be shared according to PI's decision.
Supporting Materials: Study Protocol
Time Frame: length of study

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Congenital Disorders of Glycosylation
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases