Ignite Creation Date:
2024-05-06 @ 9:40 AM
Last Modification Date:
2024-10-26 @ 12:17 PM
Study NCT ID:
NCT03041363
Status:
COMPLETED
Last Update Posted:
2022-12-22
First Post:
2017-01-31
Brief Title:
Treatment Development of Triheptanoin G1D
Sponsor:
Juan Pascual
Organization:
University of Texas Southwestern Medical Center
Study Overview
Official Title:
Treatment Development of Triheptanoin C7 for Glucose Transporter Type I Deficiency G1D A Phase I Maximum Tolerable Dose Trial
Status:
COMPLETED
Status Verified Date:
2022-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 33 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 ie 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 Childrens 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
Triheptanoin a triglyceride oil containing three odd-carbon chain-length fatty acids ie 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 Childrens 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R01NS094257 | NIH | None | httpsreporternihgovquickSearchR01NS094257 |