IRCT20131012014988N8招募中Unknown
Evaluation of the effecacy and safety of Risdiplam and Nusinersen in patients with Spinal Muscular Atrophy ??(SMA)
适应症
试验速览
- 阶段
- Unknown
- 状态
- 招募中
- 入组人数
- 40
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- no limit 至 10 years（—）
- 性别
- All
入选标准
- •Definitive genetic diagnosis of homozygous gene mutation or deletion(5q SMA)
- •Patients with equal or less than 10 years old
- •SMN2 copy number is 2 to 4
排除标准
- •Active infection requiring systemic antibiotic or antiviral treatment up to 2 weeks after recovery
- •Existence of concomitant incurable diseases (history of asphyxia, seizures, severe liver, cardiovascular, and endocrine envolvement)
- •Liver failure (due to the possibility of increased drug levels) (Child Pugh class C)
- •receiving metformin (for risdiplam)
- •Lactation and pregnancy
- •History of recent eye diseases (uncontrolled glaucoma) (for Risdiplam)
- •Existence of severe kyphoscoliosis or corrective surgery (for Nocinersen)
- •Having a CNS shunt and CSF catheter (for Nocinersen)
- •History of bacterial meningitis (for nosinersen)
研究者
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