NCT03041363

Treatment Development of Triheptanoin (G1D)

Treatment Development of Triheptanoin (C7) for Glucose Transporter Type I Deficiency (G1D): A Phase I Maximum Tolerable Dose Trial

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Juan Pascual · Academic / Other
• Sex: All
• Age: 2 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

To determine the maximum tolerated dose (MTD), as a percentage of calories consumed, of triheptanoin (C7 oil; C7) in a pediatric and adult patient population genetically diagnosed with glucose transporter type 1 deficiency disorder (G1D).

Detailed description

The trial will use an open-label, standard 3+3 phase I design for determining the MTD of orally-administered C7 in G1D. Triheptanoin: a triglyceride oil containing three odd-carbon chain-length fatty acids (i.e., a triglyceride of 7-carbon heptanoic acid). Triheptanoin will be taken 4 times per day (approximately every 6 hours) by mouth. it is dosed 4 times per day, divided evenly, and the total C7 daily dose will re-place 40% or 45% (depending on group) of the daily caloric intake from fat in the usual diet, based on current protocol guidelines. The oil should be taken approximately one hour before meals, and will be mixed with fat-free, sugar-free yogurt or pudding for administration. Up to thirty-six subjects will be enrolled in a 10-day maximum tolerable dose trial of C7. Initiation of C7 dosing will be conducted in the Children's Medical Center Dallas ambulatory Care Pavilion neurology Clinic. Subjects will be provided with C7 oil to take over the 7 days of administration. Subjects will not be required to stop other medications. Subjects will be directed to maintain their usual medications, including rescue seizure medications, as necessary for the course of the study. Subjects may have any clinical medical records transferred back to their referring physician at completion of the study.

Conditions

• Epilepsy
• GLUT1DS1
• 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 administered for 7 days 4 times daily.

Timeline

• Start date: 2017-03-29
• Primary completion: 2017-12-22
• Completion: 2017-12-22
• First posted: 2017-02-02
• Last updated: 2022-12-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study