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

ConditionSpinal Muscular Atrophy

Trial statusNot yet recruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age2+

SponsorNovartis Pharmaceuticals

About this trial

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

Qualifiers

None

Disqualifiers

None

Trial design

Treatments tested in this trial

Not listed

Trial groups

80 Participants

are grouped into 1 trial group

Locations

This trial has no locations

Sponsors and collaborators

Novartis Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov