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

Study Identifier:
COAV101A1US09
ClinicalTrials.gov Identifier:
NCT07403214
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Novartis Pharmaceuticals
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    Jun 2025 - Jun 2025
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion 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.

    Protocol 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.

    Trial Locations

    Location
    Status
    Location
    Novartis
    East Hanover, New Jersey, United States, 07936
    Status
    N/A

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