Treatment Development of Triheptanoin (C7) for Glucose Transporter Type I Deficiency (G1D): A Phase I Maximum Tolerable Dose Trial
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Maximum tolerated dose trial
研究概览
简要总结
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).
详细描述
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. Subjects 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 directed 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.
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
- 2 Years 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 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.
排除标准
- •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
- •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.
- •Allergy/sensitivity to C
- •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 1 parent or legal guardian/representative to give written informed consent.
研究组 & 干预措施
Triheptanoin
Dose 1 C7 administered as 40% daily caloric intake. Dose 2. C7 administered as 45% daily caloric intake.
干预措施: Triheptanoin (Drug)
结局指标
主要结局
Maximum tolerated dose trial
时间窗: medication taken daily for 7 days.
To determine the MTD as a percentage of calories consumed in pediatric and adult patient population.
次要结局
未报告次要终点
研究者
Juan Pascual
PROFESSOR
University of Texas Southwestern Medical Center