Experience of Using Lipano in Children With LCHAD (Long Chain Fatty Acid Oxidation Disorder)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- Gastrointestinal tolerance: questionnaire
研究概览
简要总结
This is a market research, observational study to evaluate the tolerability and acceptability of an MCT based formula for infants and young people with long chain fatty acid oxidation disorder from birth to 18 months. Patients with a confirmed diagnosis of a fatty acid oxidation disorder, which requires a specialist diet including MCT will be included in this 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
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of a fatty acid oxidation disorder requiring a ketogenic or a specialist diet as part of their treatment, which includes MCT.
- •Patients from birth to 18 months
- •Written informed consent obtained from patient or parental caregiver
排除标准
- •Presence of serious concurrent illness
- •Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- •Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
- •Any patients having taken antibiotics over the previous 2 weeks leading up to the study
- •Patients over 18 months of age
结局指标
主要结局
Gastrointestinal tolerance: questionnaire
时间窗: 90 days
Daily questionnaire detailing any GI symptoms, severity and change from usual The collection of daily data about the gastrointestinal tolerance of the product
Product compliance
时间窗: 90 days
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Product palatability
时间窗: 90 days
Questionnaire data captured to evaluate taste
Product acceptability
时间窗: 90 days
Brief tick-box questionnaire on overall liking and acceptability of product
次要结局
- Biochemistry stability - creatine kinase(90 days)
- Biochemistry stability - liver enzymes(90 days)
- Anthropometry 1(90 days)
- Anthropometry 2(90 days)