Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03301532|
Recruitment Status : Active, not recruiting
First Posted : October 4, 2017
Last Update Posted : December 23, 2022
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|Condition or disease||Intervention/treatment||Phase|
|GLUT1DS1||Drug: Triheptanoin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency|
|Actual Study Start Date :||June 5, 2018|
|Estimated Primary Completion Date :||June 18, 2023|
|Estimated Study Completion Date :||July 5, 2024|
Experimental: Patients on a ketogenic diet
This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil.
Dietary supplementation with triheptanoin
Other Name: C7 oil
- Ketosis [ Time Frame: 3 years ]Changes in blood betahydroxybutyric acid level.
- EEG [ Time Frame: 3 years ]Changes in abnormal EEG discharges.
- Seizure rate [ Time Frame: 3 years ]Changes in observable seizure numbers.
- Glycemia [ Time Frame: 3 years ]Changes in blood glucose levels.
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|Ages Eligible for Study:||30 Months to 35 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
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): NCT03301532
|United States, Texas|
|Dallas, Texas, United States, 75390|
|Principal Investigator:||Juan Pascual, M.D.||Study Principal Investigator|
|Responsible Party:||Juan Pascual, Professor of Medicine, University of Texas Southwestern Medical Center|
|Other Study ID Numbers:||
R01NS094257-01A1 ( U.S. NIH Grant/Contract )
|First Posted:||October 4, 2017 Key Record Dates|
|Last Update Posted:||December 23, 2022|
|Last Verified:||December 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
G1D, glucose, transporter