NCT04503213Enrolling By Invitation不适用
Longitudinal Assessment of CSF1R-Related Leukoencephalopathy Following Stem Cell Transplantation
适应症
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- Mayo Clinic
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Improvement in cognitive and motor function
研究概览
简要总结
The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.
详细描述
The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.
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
- •≥ 18 years of age.
- •Genetic confirmation of a mutation in the CSF1R gene.
- •Diagnosis of CSF1R-related leukoencephalopathy.
- •Anticipated to undergo haematopoietic stem cell transplantation (HSCT).
排除标准
- •Concurrent diagnoses that may confound neuropsychological testing; e.g., major hearing/visual impairment.
- •Concurrent diagnoses that may confound ambulatory measurements; e.g., amputee.
- •Inability to undergo magnetic resonance imaging (MRI); e.g., MR-incompatible implant.
结局指标
主要结局
Improvement in cognitive and motor function
时间窗: Through study completion, approximately 5 years
Number of participants to demonstrate stability/improvements in cognitive and motor function by detailed clinical assessment and radiographic markers of disease.
次要结局
未报告次要终点
研究者
Zbigniew K. Wszolek
Principal Investigator
Mayo Clinic
研究点 (1)
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