Actively Recruiting
A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome
Led by Oregon Health and Science University · Updated on 2026-05-13
16
Participants Needed
1
Research Sites
64 weeks
Total Duration
On this page
Sponsors
O
Oregon Health and Science University
Lead Sponsor
G
Glut1 Deficiency Foundation
Collaborating Sponsor
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Confirmed diagnosis of GLUT1 deficiency syndrome by at least two of the following: molecular genetic test showing a pathogenic or likely pathogenic variant in SLC2A1; documented low cerebrospinal fluid glucose with a CSF:blood glucose ratio of 0.6 or less; clinical features consistent with GLUT1DS such as epilepsy, movement disorders, ataxia, intellectual disability, or speech difficulties
- Presence of ataxia
You will not qualify if you...
- Unable to swallow liquids
- Change in neurological medications or dosages within the past 90 days
- Use of fucose- or mannose-containing supplements within one year before enrollment
- Presence of liver, kidney, blood, or metabolic disorders as shown by previous diagnosis or specific laboratory tests including hepatic impairment, low kidney function (eGFR below 60), hemoglobin A1c above 6.5%, low hemoglobin or platelet counts
- Pregnant, breastfeeding, or planning pregnancy within one year of enrollment
- Participation in another investigational drug trial for GLUT1 deficiency syndrome within one year of enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Oregon Health and Science University
Portland, Oregon, United States, 97239
Actively Recruiting
Research Team
C
Celena Byerlee-Dixon
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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