Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

• Register: ClinicalTrials.gov
• Last refreshed on: 9 January 2023
• Main ID: NCT03301532
• Date of registration: 29/08/2017
• Prospective Registration: Yes
• Primary sponsor: University of Texas Southwestern Medical Center
• Public title: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
• Scientific title: Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency
• Date of first enrolment: June 5, 2018
• Target sample size: 10
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03301532
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Juan Pascual, M.D.
• Affiliation: Study Principal Investigator

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical
genotyping at a CLIA-certified laboratory.

- Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have
taken place for 2 months). The initiation of a ketogenic diet is previous to - and
thus is not part of this study.

- Males and females 30 months to 35 years and 11 months old inclusive.

Exclusion Criteria:

- Subjects with evidence of independent, unrelated metabolic and/or genetic disease.

- Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome,
crohn's disease, or colitis that could increase the subject's risk of developing
diarrhea or stomach pain.

- Subjects with a BMI (body mass index) greater than or equal to 30.

- Subjects currently not on ketogenic diet.

- 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 use of triheptanoin less than 1 month prior to study initiation.

- Treatment with medium chain triglycerides in the last 24 hours.

- 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 subject or legal guardian/representative to give written
informed consent, or assent for children age 10-17,

- Addition of a new antiseizure drug in the previous 3 months.



Age minimum: 30 Months
Age maximum: 35 Years
Gender: All

Health Condition(s) or Problem(s) studied

GLUT1DS1

Intervention(s)

Drug: Triheptanoin

Primary Outcome(s)

Ketosis [Time Frame: 3 years]

Secondary Outcome(s)

Glycemia [Time Frame: 3 years]

Seizure rate [Time Frame: 3 years]

EEG [Time Frame: 3 years]

Secondary ID(s)

STU 102015-091

R01NS094257-01A1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institute of Neurological Disorders and Stroke (NINDS)