Clinical Trial

A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

Completed
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Summary
The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world. The DMTs included onasemnogene abeparvovec-xioi (OA), nusinersen, and risdiplam. This study was conducted using data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.
Trial Details
NCT Number NCT07403214
Lead Sponsor Novartis Pharmaceuticals
Conditions Muscular Atrophy, Spinal
Enrollment 4,805 participants
Start Date 2025-06-04
Primary Completion 2025-06-30 (estimated)
Study Completion 2025-06-30 (estimated)
Updated on ClinicalTrials.gov 2026-02-11