Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 126
- 试验地点
- 2
- 主要终点
- Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels
研究概览
简要总结
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.
II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.
详细描述
PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.
Patients are followed monthly for 6 months, then every 3 months until they reach the age of 13 years or death.
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
- 18 Months 至 8 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Glyceryl trierucate/glyceryl trioleate
Treatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial
干预措施: glyceryl trierucate/glyceryl trioleate (Drug)
结局指标
主要结局
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels
时间窗: Baseline, an average of 10 years, up to age 13
we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.
次要结局
- Number of Participants With T2 MRI Abnormality(10 years)