Study of L-fucose for GLUT1 Deficiency Syndrome
This study is looking at whether L-fucose can help people with GLUT1 Deficiency Syndrome (GLUT1DS). GLUT1DS is a condition that affects how the brain uses sugar. Researchers want to see if taking L-fucose, compared to a placebo (an inactive substance that looks like the real drug), can improve symptoms like problems with movement and coordination. You would take either L-fucose or the placebo by mouth three times a day. The study will measure changes in your movement and coordination using specific scales after 24 weeks. To join, you must be at least 18 years old and have a confirmed diagnosis of GLUT1DS. The study is currently unclear on its recruitment status and plans to enroll 16 participants.
- Study design
- This is a single-center, randomized, double-blind, placebo-controlled, cross-over study. This means participants will be randomly assigned to receive either L-fucose or a placebo, and neither you nor the study staff will know which you are receiving.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary outcomes are measured at 24 weeks, suggesting follow-up for at least this duration.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Rodrigo T. Starosta, MD, PhD · STUDY_DIRECTOR · Oregon Health and Science University
Who to contact
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What this trial measures
- SARA (Scale for the Assessment and Rating of Ataxia) Score24 weeks
Severity of ataxia and cerebellar involvement as measured by the SARA clinical scales. This score ranges from 0 (no ataxia) to 40 (most severe ataxia)
- Modified SARA (Scale for the Assessment and Rating of Ataxia) score24 weeks
This modified score suggested by the FDA rates severity of ataxia from 0 (no ataxia) to 16 (most severe ataxia)
- ICARS (International Cooperative Ataxia Rating Scale) Score24 weeks
This scale score the severity of ataxia and other cerebellar findings from 0 (no compromise) to 100 (maximal impairment)
- Safety labs: hemoglobin24 weeks
Changes in levels of hemoglobin in g/dL
- Safety labs: white blood cell count24 weeks
Changes in white blood cell counts as measured in cells/mm3
- Safety labs: platelet count24 weeks
Changes in platelet counts measured as cells/mm3
- Safety labs: lactate dehydrogenase24 weeks
Changes in lactate dehydrogenase (LDH) levels measured as U/L
- Safety labs: alanine-aminotransferase24 weeks
Changes in alanine-aminotransferase (ALT) measured as U/L
- Safety labs: aspartate-aminotransferase24 weeks
Changes in aspartate-aminotransferase (AST) measured as U/L
- Safety labs: gamma-glutamyltransferase24 weeks
Changes in gamma-glutamyltransferase (GGT) measured as U/L
- Safety labs: serum creatinine24 weeks
Changes in serum creatinine measured as mg/dL
- Safety labs: blood urea nitrogen24 weeks
Changes in blood urea nitrogen (BUN) measured as mg/dL
- Safety labs: serum sodium24 weeks
Changes in serum sodium (Na) as measured in mmol/L
- Safety labs: serum potassium24 weeks
Changes in serum potassium (K) measured as mmol/L
- Safety labs: serum chloride24 weeks
Changes in serum chloride (Cl) measured as mmol/L
- Safety labs: serum calcium24 weeks
Changes in serum calcium (Ca) measured as mmol/L
- Safety labs: serum bicarbonate24 weeks
Changes in serum bicarbonate/carbonate measured as mmol/L
- Subject-reported adverse events24 weeks
Rate and character (including standardized severity) of adverse events as reported by the study subjects