A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy
试验速览
- 阶段
- 不适用
- 入组人数
- 100
- 试验地点
- 3
- 主要终点
- Loes score
研究概览
简要总结
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to patient treatment are necessary.
详细描述
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to the patient treatment are necessary.
Patients with X-ALD will be recruited and invited to attend a baseline visit. After eligible patients are enrolled, retrospective and baseline data will be collected. After enrollment, patients will be seen approximately every 6 months until they meet withdrawal criteria or the Sponsor terminates the study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 13 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent by patient's parent(s)/legally acceptable representative(s). In addition, signed children's assent form according to local requirements.
- •Males patients 2-13 years of age with a confirmed diagnosis of X-ALD that fall into one of the following:
- •Asymptomatic patients without MRI evidence of cerebral involvement
- •Patients with MRI evidence of cerebral involvement (Loes score ≥0.5) with or without clinical symptoms
- •Patient who have HSCT within 3 months from enrollment
排除标准
- •Patients who are 14 years of age or older
- •Patients who are in a vegetative state
- •Patients (or their guardians) who are unwilling or unable to comply with the study procedures
- •Patients who received HSCT more than 3 months before enrollment
结局指标
主要结局
Loes score
时间窗: baseline and 24 weeks
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Neurological symptoms
时间窗: baseline and 24 weeks
Development of new neurological symptoms throughout the study
Plasma VLCFA levels
时间窗: baseline and 24 weeks
Change from baseline plasma VLCFA levels
次要结局
未报告次要终点