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NCT07767890 · ClinicalTrials.gov record · last posted 2026-08-17

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

StatusNot Yet Recruiting
PhaseNot specified
Started2026-10-30
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from the ClinicalTrials.gov record
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.
Trial Details
NCT Number NCT07767890
Lead Sponsor Novartis Pharmaceuticals
Conditions Spinal Muscular Atrophy
Enrollment 80 participants
Start Date 2026-10-30
Primary Completion 2031-03-31 (estimated)
Study Completion 2031-03-31 (estimated)
Updated on ClinicalTrials.gov 2026-08-17