Trials / Completed
CompletedNCT07403214
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 4,805 (actual)
- Sponsor
- Novartis Pharmaceuticals · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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.
Conditions
Timeline
- Start date
- 2025-06-04
- Primary completion
- 2025-06-30
- Completion
- 2025-06-30
- First posted
- 2026-02-11
- Last updated
- 2026-02-11
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT07403214. Inclusion in this directory is not an endorsement.