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A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients

2026年9月14日 更新者：Novartis Pharmaceuticals

Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)

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.

研究概览

地位

尚未招聘

条件

研究类型

观察性的

注册 (估计的)

80

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

研究联系人备份

  • 姓名：Novartis Pharmaceuticals

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 孩子
  • 成人
  • 年长者

接受健康志愿者

不

取样方法

非概率样本

研究人群

SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.

描述

Inclusion criteria:

• All patients treated with onasemnogene abeparvovec.

Exclusion criteria:

• Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

队列和干预

团体/队列
Onasemnogene Abeparvovec Intrathecal Group
SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
大体时间：Up to approximately 4 years and 5 months
Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs
大体时间：Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
大体时间：Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs
大体时间：Up to approximately 4 years and 5 months
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
大体时间：Up to approximately 4 years and 5 months
Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
大体时间：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.
Up to approximately 4 years and 5 months

次要结果测量

结果测量
措施说明
大体时间
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score
大体时间：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
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
大体时间：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

RULM is a tool used to assess upper limb function in SMA patients. It consists of 20 items in total. The first entry item classifies overall functional ability and is not scored. The remaining 19 tasks are scored with 1 task scored as can/cannot (1 is the highest score) and 18 tasks rated on a 3-point scale as follows:

  • 0 - unable to perform the task
  • 1 - able to perform the task with some modification
  • 2 - able to perform the task with no difficulty The total RULM score ranges from 0 to 37 with higher scores indicating better upper limb function.
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
大体时间：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
CHOP-INTEND is a tool used to measure the motor abilities in infants with SMA. It consists of 16 items scored on a scale from 0 (no response/unable to perform the movement) to 4 (complete response/able to perform the task). CHOP-Intend total score ranges from 0 to 64 with higher scores indicating higher motor function.
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
大体时间：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

The CGI-S scale is a physician-rated measure used to assess the severity of a patient's illness. Severity of illness is assessed with consideration for the overall clinical experience of patients of the same age. The CGI-S scores range from:

  • 0 = Not evaluated
  • 1 = Normal, not at all ill
  • 2 = Borderline normal and ill
  • 3 = Mildly ill
  • 4 = Moderately ill
  • 5 = Markedly ill
  • 6 = Severely ill
  • 7 = Among the most extremely ill
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
大体时间：6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

The CGI-I scale is a physician-rated measure used to assess a patient's overall clinical status. The patient's overall clinical status is scored on a 7-point scale of the extent of change relative to before treatment initiation as follows:

  • 1 = Very much improved
  • 2 = Much improved
  • 3 = Minimally improved
  • 4 = No change
  • 5 = Minimally worse
  • 6 = Much worse
  • 7 = Very much worse Number of patients and their 95% confidence intervals (CIs) will be calculated for two categories: improved (CGI-I score: 1 to 3) and unchanged or worsened (CGI-I score: 4 to 7).
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
大体时间：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

Developmental milestones include:

  • Sitting without support
  • Hands-and-knees crawling
  • Standing with assistance
  • Walking with assistance
  • Standing alone
  • Walking alone
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
大体时间：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Novartis Pharmaceuticals

调查人员

  • 研究主任：Novartis Pharmaceuticals、Novartis Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年10月30日

初级完成 (估计的)

2031年3月31日

研究完成 (估计的)

2031年3月31日

研究注册日期

首次提交

2026年8月11日

首先提交符合 QC 标准的

2026年8月11日

首次发布 (实际的)

2026年8月17日

研究记录更新

最后更新发布 (实际的)

2026年9月17日

上次提交的符合 QC 标准的更新

2026年9月14日

最后验证

2026年9月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • COAV101B1401

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

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