CTRI/2021/10/037251招募中Unknown
Observational, Non-Interventional, Post-marketing surveillance study of Risdiplam in Spinal Muscular Atrophy patients in India.
试验速览
- 阶段
- Unknown
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 7
- 主要终点
- The primary objective of the study is to assess the safety and effectiveness of risdiplam for the treatment of SMA in patients of age 2 months and older in India.
研究概览
简要总结
| This is a non-interventional, single-arm, observational study with primary prospective data collection (NIS PDC) and retrospective collection of prior medical/treatment history data from medical records. Study is designed to evaluate the safety and effectiveness of risdiplam for the treatment of SMA patients in the routine clinical setting. All treatment choices in this study will be at the discretion of the treating physician according to local standard of care or best practice and are independent of participation in this study. No additional laboratory or other diagnostic tests will be performed on patients due to their participation in this study. No study-specific site visits are mandated by the study protocol. |
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 2.00 Month(s) 至 60.00 Year(s)（—）
- 性别
- All
入选标准
- •Have signed the informed consent/assent (as applicable).
- •In the case of minors, a parent or authorized adult will be required to sign the Informed Consent Form on behalf of the patient.
- •The informed consent/assent will be obtained in accordance with local requirements
- •Patients aged ≥2 months at enrollment (for preterm infants this is the corrected age)
- •Confirmed diagnosis of 5q autosomal recessive SMA including genetic confirmation of homozygous deletion or compound heterozygosity that is predictive of loss of function of the survival motor neuron 1 (SMN1) gene and/or SMN2 gene
- •Clinical history, signs or symptoms attributable to SMA in patients of age 2 months and older 5.
排除标准
- 未提供
结局指标
主要结局
The primary objective of the study is to assess the safety and effectiveness of risdiplam for the treatment of SMA in patients of age 2 months and older in India.
时间窗: Three years
次要结局
- To describe the profile of SMA patients (2 months of age and older) in India based on demographics and familial, clinical, and genetic characteristics.(Three years)
研究者
研究点 (7)
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