A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome
A Phase II Randomized, Double-blind, Placebo-controlled, Cross-over Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome
Oregon Health and Science University
16 participants
May 5, 2026
INTERVENTIONAL
Conditions
Summary
This is a single-center, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of L-fucose supplementation in subjects with GLUT1 deficiency syndrome (GLUT1DS).
Eligibility
Inclusion Criteria3
- Age ≥ 18 years
- 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)
- Presence of ataxia
Exclusion Criteria11
- Inability to swallow liquids
- Change in neurological medications (either medication itself or medication dosages) in the past 90 days
- Use of fucose- or mannose-containing supplements within one year of enrollment
- 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):
- Any degree of hepatic impairment based on the Child-Pugh classification
- eGFR (as measured by serum creatinine or cystatin C) < 60 mg/min/1.73m2
- Hemoglobin A1c > 6.5%
- Hemoglobin level below the lower limit of normal (LLN) for sex and age
- Platelet counts below the LLN for sex and age
- Subjects who are pregnant, breastfeeding, or planning to become pregnant within one year of enrollment
- Enrollment in an investigational new drug trial for G1DS within one year of enrollment
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Interventions
L-fucose will be administered as 500 mg/kg to a maximum of 10 g three times per day by mouth.
Placebo will be composed of micro-cellulose powder with a small amount of Stevia for taste mimicking, to be taken at 500 mg/kg for a maximum of 10 g three times per day by mouth.
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07432490