NCT04482049已完成不适用
A Prospective, Multicenter, Non-interventional Study to Investigate the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders (FAOD)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 61
- 试验地点
- 15
- 主要终点
- To evaluate change in function as measured by 12 minute walk test in adult subjects with long chain FAOD
研究概览
简要总结
The purpose of the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD).
详细描述
In this study there will be no drug intervention. The study will include a Baseline visit and a follow up visit scheduled at Month 4. At these visits medical history, safety assessments, concomitant medications, exercise tests and quality of life questionnaire data will be collected.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A genetically confirmed diagnosis of one of the following:
- •Carnitine palmitoyltransferase 2 deficiency
- •Very long-chain Acyl-CoA dehydrogenase deficiency
- •Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
- •Trifunctional protein deficiency
- •A stable treatment regimen for at least 30 days
- •Ambulatory and able to perform the study exercise test, using walking aids if necessary
- •Willing and able to personally sign and date an informed consent document indicating that the subject has been informed of all pertinent aspects of the study
排除标准
- •Unstable or poorly controlled disease as determined by one or more of the following:
- •Presence of symptoms of acute rhabdomyolysis with clinically significant elevations in serum CK
- •Evidence of acute crisis from their underlying disease
- •Currently taking a PPAR agonist
- •Have motor abnormalities other than those related to the fatty acid oxidation disorder that could interfere with the study procedures, as determine by the investigator
- •Evidence of significant concomitant medical or psychiatric disease that in the opinion of the Investigator may interfere with the conduct or safety of this study
- •Pregnant or nursing females
结局指标
主要结局
To evaluate change in function as measured by 12 minute walk test in adult subjects with long chain FAOD
时间窗: Week 16
Change from baseline in 12MWT
次要结局
- To evaluate change in symptoms related to FAOD using a newly developed muscle symptom questionnaire specifically designed for adult patients with long chain FAOD(Week 16)
研究者
研究点 (15)
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