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

Completed

Conditions studied: Muscular Atrophy, Spinal

In brief

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.

Key facts

Study ID

NCT07403214

Run by

Novartis Pharmaceuticals

People needed

4805

Starts

2025-06-04

Expected to finish

2025-06-30

Last updated by the study team

2026-02-11

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients with ≥1 SMA diagnosis at any time (International Classification of Disease, Tenth Revision, Clinical Modification [ICD-10-CM] codes: G12.0, G12.1, G12.9).
• Patients with ≥1 record of OA, nusinersen, or risdiplam based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC).
• Patients who received at least 2 of the 3 DMTs, with the first DMT initiated on or after 26 May 2019, when at least 2 DMTs were available. The initiation of the first DMT was defined as the index date and the first DMT was defined as the index DMT.
• Patients with SMA types 1, 2, or 3.
• Patients with ≥1 quarter of clinical activities within 1 year prior to the index quarter (for patients with SMA type 2 and 3 only).
• Patients with ≥1 quarter of clinical activities any time after the index quarter, unless death occurred.

You may not qualify if…

• Patients with SMA type 4.
• Patients with claims of multiple DMTs in their birth year.

Where it is running

• Novartis — East Hanover, New Jersey, United States

Full record on ClinicalTrials.gov

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