CompletedNot Applicable

Using D-Galactose as a Food Supplement in Congenital Disorders of Glycosylation

United States8 participantsStarted 2014-07

Plain-language summary

The goal of this study is to better characterize the metabolic alterations and sugar structure alterations (glycosylation abnormalities) in patients diagnosed with Congenital Disorders of Glycosylation. The investigators aim to assess the safety and tolerability of oral galactose treatment in a small pilot group of Congenital Disorders of Glycosylation patients. The investigators will also determine the relationship between simple milk sugar intake (galactose dose) in the diet and the blood and urine markers of protein glycosylation abnormalities.

Who can participate

Age range

21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Biochemically and genetically proven Congenital Disorders of Glycosylation. Exclusion Criteria: * Any of the following conditions: * Aldolase B Deficiency * Galactosemia (unable to process galactose) * Hemolytic uremic syndrome * Severe anemia * Diagnosis of intellectual disability or developmental delay * Galactose Intolerance * Has previously experienced any of the following severe side effects from oral galactose: * Diarrhea * Vomiting * Constipation * Galactosuria (Galactose in the urine) * Increased liver glycogen storage.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Metabolic Function

Timeframe: 18 weeks

Trial details

NCT IDNCT02955264

SponsorTulane University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-11

View on ClinicalTrials.gov More Congenital Disorders of Glycosylation trials