Last Update: Oct 22, 2026
Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)
ClinicalTrials.gov Identifier:
NCT07767890
Novartis Reference Number:COAV101B1401
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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.

Spinal Muscular Atrophy
Recruiting
80
Oct 07, 2026
Mar 31, 2031
All
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).

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

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