Congenital Disorder of GlycosylationNCT04198987
Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation
An observational study of Congenital Disorder of Glycosylation, run by Mayo Clinic at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Disorder of Glycosylation
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Dec 2018to 11 Jun 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
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.
Who may be able to join
- Patient has a biochemically and genetically proven congenital disorder of glycosylation
- Patient is receiving (or planning to receive) oral simple sugar supplementation
Who cannot join
- Aldolase B deficiency
- Galactosemia
- Hemolytic uremic syndrome
- Severe anemia
- Galactose intolerance
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
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).
Read the 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.
- Conditions
- Congenital Disorder of Glycosylation
- Medical subject headings
- Congenital Disorders of Glycosylation
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: length of study, up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2019
- Study start6 Dec 2018
- Primary completion11 Jun 2025
- Study completion11 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Disorder of GlycosylationStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, David R. Deyle, Mayo Clinic
- Investigators
- David Deyle, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04198987
- Sponsor's protocol ID
- 18-007276
- Status last verified
- Jan 2025
- First submitted
- 8 Dec 2019
- Last updated
- 1 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Data will be shared according to PI's decision.
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation
- ClinicalTrials.gov ID
- NCT04198987
- Acronym / sponsor ID
- 18-007276
- Conditions
- Congenital Disorder of Glycosylation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 6 Dec 2018 (actual)
- Primary completion
- 11 Jun 2025 (actual)
- Study completion
- 11 Jun 2025 (actual)
- First posted
- 13 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- Patient has a biochemically and genetically proven congenital disorder of glycosylation
- Patient is receiving (or planning to receive) oral simple sugar supplementation
Key exclusion criteria (5 listed)
- Aldolase B deficiency
- Galactosemia
- Hemolytic uremic syndrome
- Severe anemia
- Galactose intolerance
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2025.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Data will be shared according to PI's decision.
Related trials for Congenital Disorder of Glycosylation
RecruitingObservational study
Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation
Congenital Disorders of Glycosylation
Enrolling by invitationPhase 2
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Active, not recruitingPhase 2/3
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
Phosphomannomutase 2 Deficiency, PMM2-CDG
RecruitingObservational study
Clinical and Basic Investigations Into Congenital Disorders of Glycosylation
Congenital Disorders of Glycosylation
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Deyle, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Disorder of Glycosylation, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measuring effects of oral monosaccharide supplements through growth measurements (time frame: length of study, up to 2 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2025.