Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Change in Ketosis (Beta-hydroxybutyrate Levels)
研究概览
简要总结
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
详细描述
This is a single site, open label proof of principle exploratory trial to investigate the compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic diet will have been previously described by the patient's treating physician independently of this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from fat, subjects, who are already tolerating over this much fat as part of their previously prescribed ketogenic diet, will replace 45% of their daily caloric intake with the triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous EEG to monitor for any potential C7 related changes in seizure before, during, and after triheptanoin oil ingestion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 30 Months 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
- •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.
研究组 & 干预措施
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.
干预措施: Triheptanoin (Drug)
结局指标
主要结局
Change in Ketosis (Beta-hydroxybutyrate Levels)
时间窗: Day 1, Day 4
Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.
次要结局
- Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate(Baseline (Day 1) - Day 4)
- Change in Glycemia(Day 1 - Day 4)
研究者
Juan Pascual
Professor of Medicine
University of Texas Southwestern Medical Center