NCT07274826进行中（未招募）不适用
Intact Potential Symptoms and Biomarker Analysis in Diagnosis of Acid Sphingomyelinase Deficiency (ASMD)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- spleen volumes measured by US expressed relative to basaline for each patient
研究概览
简要总结
Acid sphingomyelinase Deficiency known as Neiman _PICK disease is a group of rare genetic diseases. This study includes analysis of clinical manifestations in patients with ASMD and investigations done for diagnosis of these patients
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •all patients diagnosed with ASMD in Sohag
排除标准
- •Patients with hepatosplenomegaly due to other cause Patients who refuse consent
结局指标
主要结局
spleen volumes measured by US expressed relative to basaline for each patient
时间窗: From basaline to month 12
Change in spleen volumes following treatment over 12months study period
次要结局
- Liver size measured by ultrasound(Basaline, weak 12,weak 24)
研究者
研究点 (1)
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