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Clinical Trials in Japan / NCT07767890
Recruiting Observational

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

NCT07767890 · tracked via the Priya Life Science Japan tracker
Sponsor
Novartis Pharmaceuticals
Phase
Observational
Started
2026-10-07
Last updated
2026-10-22

Condition(s) studied

Spinal Muscular Atrophy

Study 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

Sex
ALL
Min age
2 Years
Max age
—
Healthy volunteers
No
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).

Primary outcome measure(s)

  • Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs) — Up to approximately 4 years and 5 months
  • Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications — Up to approximately 4 years and 5 months
    Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.
  • Incidence of Serious Adverse Events and ADRs — Up to approximately 4 years and 5 months
    Incidence based on the person-year method will be calculated.
  • Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications — Up to approximately 4 years and 5 months
    Incidence based on the person-year method will be calculated.
  • Number of Patients by Time to Onset of Serious Adverse Events and ADRs — Up to approximately 4 years and 5 months
  • Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications — Up to approximately 4 years and 5 months

Trial sites (4)

FacilityCityRegionStatus
Novartis Investigative Site Toyoake Aichi-ken Recruiting
Novartis Investigative Site Fukuoka Fukuoka Recruiting
Novartis Investigative Site Kyoto Kyoto Recruiting
Novartis Investigative Site Kodaira Tokyo Recruiting

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Other trials for the same condition

A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07767890 on ClinicalTrials.gov ↗ ← All trials in Japan