CompletedN/AOther
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
Sponsored by Novartis Pharmaceuticals
NCT ID
NCT07403214
Target Enrollment
4,805 participants
Start Date
2025-06-04
Est. Completion
2025-06-30
About This Study
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 Studied
Eligibility
Healthy Volunteers:No
View full eligibility criteria
Inclusion criteria * 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. Exclusion criteria * Patients with SMA type 4. * Patients with claims of multiple DMTs in their birth year.
Study Locations (1)
Novartis
East Hanover, New Jersey, United States