NCT07767890Recruiting
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)
Source: ClinicalTrials.gov · Last updated 22 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 80 (estimated)
- First submitted
- 11 Aug 2026
- Start date
- 7 Oct 2026
- Primary completion date
- 31 Mar 2031
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07767890
What you commit to
- Study type: Observational
- Planned participants: 80
- From 7 Oct 2026 to the primary completion date, 31 Mar 2031
- 4 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).
Who the study is looking for
- All patients treated with onasemnogene abeparvovec.
Age in the registry: 2 years to - · Sex: All · Does not accept healthy volunteers
Sites (4)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Fukuoka | Japan | Recruiting |
| - Coordinator of this site? Confirm its status | Kodaira | Japan | Recruiting |
| - Coordinator of this site? Confirm its status | Kyoto | Japan | Recruiting |
| - Coordinator of this site? Confirm its status | Toyoake | Japan | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.