Intrathecal Zolgensma in spinal muscular atrophy patients in Japan

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a non-interventional, observational study conducted in Japan to collect real-world safety and effectiveness data for intrathecal onasemnogene abeparvovec in spinal muscular atrophy patients. No specific treatment protocol or visit schedule is imposed; patients are treated per the Japan package insert. Data are gathered via case registration forms completed by investigators at participating sites. The study includes all patients treated with onasemnogene abeparvovec, with exclusions for off-label or unapproved indications under applicable regulations. Primary insights come from routine clinical practice across three Novartis investigative sites in Japan.

Why it matters

Who can join: the full rules

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).

Where it's running

• Novartis Investigative Site
  Toyoake, Aichi-ken, Japan Recruiting
• Novartis Investigative Site
  Fukuoka, Fukuoka, Japan Recruiting
• Novartis Investigative Site
  Kyoto, Kyoto, Japan Recruiting
• Novartis Investigative Site
  Kodaira, Tokyo, Japan Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Understanding fatigue in patients with spinal muscular atrophy treated with nusinersen

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.