Treatment Development of Triheptanoin (G1D)
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|ClinicalTrials.gov Identifier: NCT03041363|
Recruitment Status : Active, not recruiting
First Posted : February 2, 2017
Last Update Posted : December 17, 2020
|Condition or disease||Intervention/treatment||Phase|
|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||Drug: Triheptanoin||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||36 participants|
|Intervention Model:||Single Group Assignment|
|Intervention 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)|
|Official Title:||Treatment Development of Triheptanoin (C7) for Glucose Transporter Type I Deficiency (G1D): A Phase I Maximum Tolerable Dose Trial|
|Actual Study Start Date :||March 29, 2017|
|Estimated Primary Completion Date :||March 29, 2022|
|Estimated Study Completion Date :||March 29, 2025|
Dose 1 C7 administered as 40% daily caloric intake. Dose 2. C7 administered as 45% daily caloric intake.
Triheptanoin will be administered for 7 days 4 times daily.
Other Name: C7
- Maximum tolerated dose trial [ Time Frame: medication taken daily for 7 days. ]To determine the MTD as a percentage of calories consumed in pediatric and adult patient population.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03041363
|United States, Texas|
|UT Southwestern Medical Center|
|Dallas, Texas, United States, 75390|
|Principal Investigator:||Juan Pascual||UT Southwestern Medical Center|