NCT07378943

A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies

Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy (SMA) Receiving Disease Modifying Therapies (DMT) - Real-world Analysis Using the Komodo Claims Data

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 4,114 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam. This study was conducted using both open and closed claims data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.

Conditions

• Muscular Atrophy, Spinal

Timeline

• Start date: 2024-11-06
• Primary completion: 2025-03-30
• Completion: 2025-03-30
• First posted: 2026-01-30
• Last updated: 2026-01-30

Locations

1 site across 1 country: United States