NCT03181399

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Dietary Treatment of Glucose Transporter Type 1 Deficiency (G1D)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: University of Texas Southwestern Medical Center · Academic / Other
• Sex: All
• Age: 24 Months – 35 Years
• Healthy volunteers: Not accepted

Summary

Forty-five subjects receiving no dietary therapy with a proven G1D diagnosis will be enrolled. To evaluate the effect of C7 supplementation of a regular diet on a EEG activity in addition to IQ, language, working memory, processing speed, emotional and behavioral functioning, ataxia, and other neuropsychological and neurological performance indices in children and adults genetically diagnosed with G1D receiving a regular diet at enrollment.

Detailed description

This is an open-label, single arm trial of orally-administered C7 in G1D. Subjects will replace a fixed percentage of their daily caloric intake (based on the results of Protocol 1) with C7 for 6 months, undergo full evaluation and discontinuation of treatment at a 6 month visit, and return for an off-treatment follow up visit 3 months after C7 oil discontinuation, for total duration of participation of 9 months. Subjects will undergo treatment initiation on a 24-hour inpatient basis. During that 24-hr inpatient treatment initiation, subjects will have continuous EEG both to monitor for real-time seizure activity (for safety) and to determine EEG changes (secondary outcome) before, during, and after treatment initiation. Subjects will undergo clinical evaluation, comprehensive blood work, ataxia scale rating, EEG, and neuropsychological testing at baseline, 6 months, and 9 months.

Conditions

• GLUT1DS1
• Epilepsy
• Glut1 Deficiency Syndrome 1, Autosomal Recessive
• Glucose Metabolism Disorders
• Glucose Transport Defect
• Glucose Transporter Type 1 Deficiency Syndrome
• Glucose Transporter Protein Type 1 Deficiency Syndrome

Interventions

Type	Name	Description
DRUG	Triheptanoin	. Triheptanoin will be taken 4 times per day (approximately every 6 hours: prior to breakfast, lunch and dinner and a mid-afternoon snack) by mouth. It is dosed 4 times per day, divided evenly.

Timeline

• Start date: 2018-04-18
• Primary completion: 2023-09-10
• Completion: 2023-09-10
• First posted: 2017-06-08
• Last updated: 2025-08-07
• Results posted: 2025-04-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study