Treatment Development of Triheptanoin (G1D)
Last updated on July 2021Recruitment
- Recruitment Status
- Active, not recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Epilepsy
- Glucose Metabolism Disorders
- Glucose Transport Defect
- Glucose Transporter Protein Type 1 Deficiency Syndrome
- Glucose Transporter Type 1 Deficiency Syndrome
- Glut1 Deficiency Syndrome 1, Autosomal Recessive
- GLUT1DS1
- Type
- Interventional
- Phase
- Phase 1
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentIntervention Model Description: The trial will use an open-label, standard 3+3 phase 1 design for determining the MTD of orally administered C7 in G1D.Masking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 2 years and 35 years
- Gender
- Both males and females
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 ...
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.
Tracking Information
- NCT #
- NCT03041363
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Investigators
- Principal Investigator: Juan Pascual UT Southwestern Medical Center