JPRN-UMIN000045717招募中未知
Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy - PRIME-SMA (Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy)
适应症
试验速览
- 阶段
- 未知
- 状态
- 招募中
- 入组人数
- 30
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 6years-old 至 ot applicable（—）
- 性别
- All
入选标准
- 未提供
排除标准
- •<Cohort A> (1) Patients who have received Nusinersen <Cohort A and Cohort B> (1) Patients who have received Risdiplam <Cohort A, Cohort B and Cohort C> (1) Patients who have received Onasemnogene abeparvovecor other SMN2 modifiers (2) Patients who are receiving valproic acid. (3) Patients who have undergone surgery for scoliosis or hip arthrodesis within 6 months before screening or are scheduled to undergo within 18 months after screening (4) Patients who require invasive ventilation or tracheostomy (5) Patients who are judged by the investigator to have a disease other than SMA that causes functional decline in the upper limbs (6) Patients who are judged by the investigator not to be suitable for this study for any reason
研究者
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