Treatment Development of Triheptanoin (G1D) (NCT03041363) | Clinical Trial Compass
CompletedPhase 1
Treatment Development of Triheptanoin (G1D)
United States12 participantsStarted 2017-03-29
Plain-language 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).
Who can participate
Age range2 Years ā 35 Years
SexALL
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Inclusion Criteria:
* Diagnosis of glucose transporter type I deficiency (G1D) confirmed by genotyping or PET scan of the brain.
* Stable on no dietary therapy other than Modified Atkins diet (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 35 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, 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.
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