A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome

Conditions

• GLUT1DS1
• Glut1 Deficiency

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• L-fucose — DRUG
  L-fucose will be administered as 500 mg/kg to a maximum of 10 g three times per day by mouth.
• Placebo — OTHER
  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.

Study Details

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).

Key Dates

Start date

May 5, 2026

Status verified

May 2026

Primary completion

Jul 31, 2027

Completion

Aug 31, 2027

Study Design

Enrollment

16 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Active Comparator: L-fucose followed by placebo
  L-fucose for 12 weeks, followed by placebo for 12 weeks.
• Placebo Comparator: Placebo followed by L-fucose
  Placebo for 12 weeks, followed by L-fucose for 12 weeks.

Primary Outcome Measure

SARA (Scale for the Assessment and Rating of Ataxia) Score [ Time Frame: 24 weeks ]

Central Contacts

• Celena Byerlee-Dixon
  503-494-7004
  [email protected]

Locations (1)

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