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NCT07767890
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
Conditions: Spinal Muscular Atrophy
Sex: All
Ages: 2 Years – N/A
Healthy volunteers: No
Enrollment: 80
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.
Eligibility Criteria
Inclusion criteria:
• All patients treated with onasemnogene abeparvovec.
Exclusion criteria:
• Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).
Source: ClinicalTrials.gov (NCT07767890). StuddyBuddy aggregates publicly available trial information.
