Safety and Effectiveness of Zolgensma Injection in Spinal Muscular Atrophy
Observes how onasemnogene abeparvovec works in people with spinal muscular atrophy in routine care.
Plain-language title generated from the registry record
Registry title: A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
Key facts
- Where
- Fukuoka, Kodaira, Kyoto, and Toyoake, JapanSee the 4 sites
Where in Japan
- FukuokaFukuoka, JapanTaking people
- KodairaKodaira, JapanTaking people
- KyotoKyoto, JapanTaking people
- ToyoakeToyoake, JapanTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharmaceuticals
- Trial code
- NCT07767890
- Record updated
- 22 October 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: All patients treated with onasemnogene abeparvovec.
Exclusion: Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).