Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 80
- 主要终点
- Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
研究概览
简要总结
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients treated with onasemnogene abeparvovec.
排除标准
- •Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).
研究组 & 干预措施
Onasemnogene Abeparvovec Intrathecal Group
SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.
结局指标
主要结局
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
时间窗: Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs
时间窗: Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
时间窗: Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Number of Patients by Time to Onset of Serious Adverse Events and ADRs
时间窗: Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
时间窗: Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
时间窗: Up to approximately 4 years and 5 months
Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.
次要结局
- Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Change From Baseline in Revised Upper Limb Module (RULM) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score(6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Number of Patients Achieving Each Developmental Milestone(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)
- Number of Patients Maintaining Each Developmental Milestone(Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months)