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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 : June 21, 2018
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
University of Texas Southwestern Medical Center

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).

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

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. Sub-jects 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 di-rected 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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: Triheptanoin
Dose 1 C7 administered as 40% daily caloric intake. Dose 2. C7 administered as 45% daily caloric intake.
Drug: Triheptanoin
Triheptanoin will be administered for 7 days 4 times daily.
Other Name: C7




Primary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of glucose transporter type I deficiency (G1D) confirmed by genotyping or PET scan of the brain.
  • Stable on no dietary therapy (i.e., on no dietary therapy for 1 month, including, but not limited to, medium chain triglyceride therapy).
  • Males and females 2 years 6 months to 17 years 11 months old, inclusive.

Exclusion Criteria:

  • Subjects with a history of life-threatening seizure episodes, including but not limited to status epilepticus and cardiac arrest.
  • Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
  • Subjects with a body mass index (BMI) greater than or equal to 30.
  • Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, Crohn's disease, or colitis, which could increase the subject's risk of developing diarrhea or stomach pain.
  • Subjects currently on dietary therapy (i.e., ketogenic diet, medium chain triglyceride-supplemented diets, Atkins diet and versions of it, low glycemic index diet, and related diets).
  • Women who are pregnant or breast-feeding may not participate.
  • Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate.
  • Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick.
  • Subjects will be asked to agree to abstinence or another form of birth control for the duration of the study.
  • Allergy/sensitivity to C7.
  • Previous treatment with C7 one month prior to enrollment.
  • Treatment with medium chain triglycerides in the last 30 days.
  • Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain disorder (such as Alzheimer's disease) that would confound assessment of cognitive changes, in the opinion of the investigator.
  • Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements.
  • Inability or unwillingness of 2 parents or legal guardians/representatives to give written informed consent.

Information from the National Library of Medicine

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


Locations
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Sponsors and Collaborators
University of Texas Southwestern Medical Center
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
Principal Investigator: Juan Pascual UT Southwestern Medical Center

Additional Information:
Publications:
Responsible Party: University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT03041363     History of Changes
Other Study ID Numbers: STU 062016-105
R01NS094257 ( U.S. NIH Grant/Contract )
First Posted: February 2, 2017    Key Record Dates
Last Update Posted: June 21, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Metabolic Diseases
Glucose Metabolism Disorders
Carbohydrate Metabolism, Inborn Errors
Glycogen Storage Disease Type I
Disease
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease