Revised August 13, 2019
NIH Funded Research Studies
Triheptanoin (C7) treatment for Glucose transporter type 1 deficiency (G1D) with or without the ketogenic diet
(two separate studies)
Email: Rare.Diseases@UTSouthwestern.edu (physicians or patients).
Location: The University of Texas Southwestern Medical Center, Dallas, Texas.
Funding agencies: National Institute of Neurological Disorders and Stroke (National Institutes of Health)
and Glut1 Deficiency Foundation
Approvals: UT Southwestern Medical Center Institutional Review Board and FDA
Principal Investigator: Juan M. Pascual, MD, PhD
Two NIH-funded studies are ongoing using food-grade C7 oil in G1D.
The purpose of the first study is to answer the question of whether C7 impacts neuropsychological test results (cognitive capacity, including attention, comprehension, memory, etc.) in G1D patients taking a regular diet.
A second study (unrelated) investigates whether C7 is compatible (i.e., can be taken) with the ketogenic diet. EEG and blood work will be part of these studies.
Patients diagnosed by DNA testing or by PET scan will be eligible for these studies. They may not be (for the first study) but must be (for the second study) taking a ketogenic diet. No patient will be asked to or should change diets just for these studies.
There will be no patient or medical insurance charges. Travel expenses up to $1,220 plus a $250 stipend for lost time are available upon submission of expense receipts after each visit is completed. Reimbursement is via prepaid credit card. Discounted local hotel rates are available.
The University of Texas Southwestern Medical Center and the NIH are non-profit institutions and hold no commercial interests in these studies.
Location: The University of Texas Southwestern Medical Center, Dallas, Texas.
Funding agencies: National Institute of Neurological Disorders and Stroke (National Institutes of Health)
and Glut1 Deficiency Foundation
Approvals: UT Southwestern Medical Center Institutional Review Board and FDA
Principal Investigator: Juan M. Pascual, MD, PhD
Two NIH-funded studies are ongoing using food-grade C7 oil in G1D.
The purpose of the first study is to answer the question of whether C7 impacts neuropsychological test results (cognitive capacity, including attention, comprehension, memory, etc.) in G1D patients taking a regular diet.
A second study (unrelated) investigates whether C7 is compatible (i.e., can be taken) with the ketogenic diet. EEG and blood work will be part of these studies.
Patients diagnosed by DNA testing or by PET scan will be eligible for these studies. They may not be (for the first study) but must be (for the second study) taking a ketogenic diet. No patient will be asked to or should change diets just for these studies.
- The first study (ClinicalTrials.gov NCT03181399) lasts 9 months (6 months on C7 and then 3 months off C7) and requires three 3-day visits to Dallas over 9 months. About 24 hrs. of the 3 days will be inpatient.
- For the second study (ClinicalTrials.gov NCT03301532), eligible patients will be previously receiving a ketogenic diet as part of their routine G1D management. This is a short (about 4-day) study. About two of the 4 days will be inpatient.
There will be no patient or medical insurance charges. Travel expenses up to $1,220 plus a $250 stipend for lost time are available upon submission of expense receipts after each visit is completed. Reimbursement is via prepaid credit card. Discounted local hotel rates are available.
The University of Texas Southwestern Medical Center and the NIH are non-profit institutions and hold no commercial interests in these studies.