Actively Recruiting
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Led by Novartis Pharmaceuticals · Updated on 2025-01-15
50
Participants Needed
2
Research Sites
775 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
CONDITIONS
Official Title
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject's parent or legal guardian has provided signed informed consent form.
- Subject has genetically confirmed Spinal Muscular Atrophy with bi-allelic mutation in SMN1 gene and clinical diagnosis of SMA Type 1 or up to 3 copies of SMN2 gene.
- Subject has been treated with Onasemnogene Abeparvovec (Zolgensma4) prior to enrolling.
- Subjects previously treated with nusinersen or risdiplam can be enrolled if not currently receiving those treatments.
- Subject and parent/guardian are willing and able to comply with phone contacts during the study.
You will not qualify if you...
- Patients currently enrolled in any interventional clinical trial other than the phase IV OFELIA trial are excluded.
- Subjects previously enrolled in any clinical trial but not currently enrolled may be included.
- Subjects who enroll in a clinical trial with pharmacological intervention during follow-up will discontinue from this study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Novartis Investigative Site
Curitiba, Paraná, Brazil, 81520-060
Actively Recruiting
2
Novartis Investigative Site
São Paulo, São Paulo, Brazil, 05403-000
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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