NCT07767890 A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
| NCT ID | NCT07767890 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Novartis Pharmaceuticals |
| Condition | Spinal Muscular Atrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 80 participants |
| Start Date | 2026-10-07 |
| Primary Completion | 2031-03-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 80 participants in total. It began in 2026-10-07 with a primary completion date of 2031-03-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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).
Contact & Investigator
Novartis Pharmaceuticals
STUDY DIRECTOR
Novartis Pharmaceuticals
Frequently Asked Questions
Who can join the NCT07767890 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Spinal Muscular Atrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07767890 currently recruiting?
Yes, NCT07767890 is actively recruiting participants. Contact the research team at novartis.email@novartis.com for enrollment information.
Where is the NCT07767890 trial being conducted?
This trial is being conducted at Toyoake, Japan, Fukuoka, Japan, Kyoto, Japan, Kodaira, Japan.
Who is sponsoring the NCT07767890 clinical trial?
NCT07767890 is sponsored by Novartis Pharmaceuticals. The principal investigator is Novartis Pharmaceuticals at Novartis Pharmaceuticals. The trial plans to enroll 80 participants.