Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Time to relapse
研究概览
简要总结
This study is being done to develop a database of pediatric patients in order to study the cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD) in pediatric patients.
详细描述
This study is being done to collect information on the natural history of NMOSD in pediatric AQP4-IgG seropositive patients. A major restriction in performing drug studies in pediatric patients with NMOSD is limited information on the course of the disease in these patients. Collecting clinical information over the course of a 1 year observational study would inform on the natural history of the disease in these patients. A repository of pediatric patients with rare diseases can increase knowledge on the natural history of the specific disease, assist in identifying appropriate patients fulfilling specified criteria for drug studies and potentially serve as a control group.
Timepoints: Baseline, 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point).
Baseline data:
- Demographics [age/sex/ethnicity],
- Clinical presentation information including date of initial diagnosis,
- Clinical phenotype
- Immunotherapy used current and past,
- Family history of autoimmune diseases,
- Serological data results
- Radiologic data as available
Self-Report Assessments will be:
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 17 Years(Child)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •AQP4 positive
- •Ability to give informed consent by patient or caregiver
排除标准
- •Inability to complete required forms via phone, mail, or email.
结局指标
主要结局
Time to relapse
时间窗: One year.
Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.
次要结局
未报告次要终点
