Last Update: Oct 22, 2026 Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy) ClinicalTrials.gov Identifier: [NCT07767890](https://clinicaltrials.gov/ct2/show/NCT07767890) Novartis Reference Number:COAV101B1401 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study Description 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. Condition Spinal Muscular Atrophy Overall Status Recruiting Number of Participants80 Start Date Oct 07, 2026 Completion Date Mar 31, 2031 Gender All Age(s) 2 Years - (Child, Adult, Older Adult) ## 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). Japan #### Novartis Investigative Site Recruiting Toyoake,Aichi-ken,4701192,Japan #### Novartis Investigative Site Recruiting Fukuoka,Fukuoka,8140180,Japan #### Novartis Investigative Site Recruiting Kyoto,Kyoto,6028566,Japan #### Novartis Investigative Site Recruiting Kodaira,Tokyo,1878551,Japan ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: