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A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
Sponsor: Novartis Pharmaceuticals
Summary
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.
Official title: Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
80
Start Date
2026-10-07
Completion Date
2031-03-31
Last Updated
2026-10-22
Healthy Volunteers
No
Conditions
Locations (4)
Novartis Investigative Site
Toyoake, Aichi-ken, Japan
Novartis Investigative Site
Fukuoka, Fukuoka, Japan
Novartis Investigative Site
Kyoto, Kyoto, Japan
Novartis Investigative Site
Kodaira, Tokyo, Japan