NCT06019637 A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
| NCT ID | NCT06019637 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Novartis Pharmaceuticals |
| Condition | Spinal Muscular Atrophies |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2023-11-22 |
| Primary Completion | 2038-09-30 |
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 50 participants in total. It began in 2023-11-22 with a primary completion date of 2038-09-30.
⚠ 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
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Eligibility Criteria
Inclusion Criteria: * Subject's parent or legal guardian has provided signed eICF. * Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene. * Subject treated\* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study. Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it. \*Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements. * Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study Exclusion Criteria: * Patients currently enrolled in any interventional clinical trial\*\* other than the phase IV OFELIA trial will be excluded from the study. * Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study. During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.
Contact & Investigator
Novartis Pharmaceuticals
STUDY DIRECTOR
Novartis Pharmaceuticals
Frequently Asked Questions
Who can join the NCT06019637 clinical trial?
This trial is open to participants of all sexes, aged 0 Years or older, up to 100 Years, studying Spinal Muscular Atrophies. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06019637 currently recruiting?
Yes, NCT06019637 is actively recruiting participants. Contact the research team at novartis.email@novartis.com for enrollment information.
Where is the NCT06019637 trial being conducted?
This trial is being conducted at Curitiba, Brazil, São Paulo, Brazil.
Who is sponsoring the NCT06019637 clinical trial?
NCT06019637 is sponsored by Novartis Pharmaceuticals. The principal investigator is Novartis Pharmaceuticals at Novartis Pharmaceuticals. The trial plans to enroll 50 participants.