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

NCT07403214 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 4805

Last updated 2026-02-11

No results posted yet for this study

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

  • Muscular Atrophy, Spinal

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-04
Primary Completion
2025-06-30
Completion
2025-06-30

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07403214 on ClinicalTrials.gov