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

NCT07767890 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 80

Last updated 2026-08-17

No results posted yet for this study

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.

Conditions

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-10-30
Primary Completion
2031-03-31
Completion
2031-03-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07767890 on ClinicalTrials.gov