Could a simple sugar help people with rare brain disorder?
NCT ID NCT07432490
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether a sugar called L-fucose can improve movement and coordination in adults with GLUT1 deficiency syndrome, a rare genetic disorder that affects the brain's energy supply. Sixteen participants will receive either fucose or a placebo for a period, then switch, so everyone gets both. The main goal is to see if fucose reduces symptoms like unsteadiness and difficulty with movement.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 16 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2026
- Expected to finish
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Aug 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Age ≥ 18 years 2. Confirmed diagnosis of GLUT1DS, including at least 2 out of the following 3: molecular genetic testing showing a pathogenic or likely pathogenic variant in SLC2A1; documented hypoglycorrhachia with a CSF:blood glucose ratio ≤ 0.6; clinical features consistent with GLUT1DS (epilepsy, movement disorders, ataxia, intellectual disability, dysarthria) 3. Presence of ataxia Exclusion Criteria: 1. Inability to swallow liquids 2. Change in neurological medications (either medication itself or medication dosages) in the past 90 days 3. Use of fucose- or mannose-containing supplements within one year of enrollment 4. Presence of hepatic, renal, hematological, or concomitant metabolic disorders, as assessed by the presence of a previous diagnosis of such disorders (for instance, chronic kidney disease, liver cirrhosis, diabetes mellitus) or by the following laboratory values, which will be considered if obtained clinically up to 90 days before enrollment (if this is not available, laboratory tests will be obtained prior to first study visit): 1. Any degree of hepatic impairment based on the Child-Pugh classification 2. eGFR (as measured by serum creatinine or cystatin C) \< 60 mg/min/1.73m2 3. Hemoglobin A1c \> 6.5% 4. Hemoglobin level below the lower limit of normal (LLN) for sex and age 5. Platelet counts below the LLN for sex and age 5. Subjects who are pregnant, breastfeeding, or planning to become pregnant within one year of enrollment 6. Enrollment in an investigational new drug trial for G1DS within one year of enrollment
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Oregon Health and Science University
RECRUITINGPortland, Oregon, 97239, United States
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