Status:
COMPLETED
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Up to 5 years
Brief Summary
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US. The study required a repeated data collection at ...
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Genetically confirmed diagnosis of SMA.
- Aged less than 5 years at index date.
- Initiated treatment with nusinersen or onasemnogene abeparvovec-xioi or risdiplam or any combination of them during the identification period.
- Availability of medical information in chart for at least 1 visit prior to initiation of treatment with the target treatment(s).
- Exclusion criteria:
- None
Exclusion
Key Trial Info
Start Date :
May 5 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 30 2022
Estimated Enrollment :
162 Patients enrolled
Trial Details
Trial ID
NCT06178393
Start Date
May 5 2022
End Date
December 30 2022
Last Update
December 21 2023
Active Locations (1)
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1
Novartis
Bannockburn, Illinois, United States, 60015