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临床试验/NCT03766347
NCT03766347已完成不适用

Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study

Mayo Clinic1 个研究点 分布在 1 个国家目标入组 100 人开始时间: 2018年2月1日最近更新:
适应症

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

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

研究者

发起方
Mayo Clinic
申办方类型
Other
责任方
Principal Investigator
主要研究者

Sean Pittock

Prinicpal Investigator

Mayo Clinic

研究点 (1)

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