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

Study Identifier:
COAV101A1US10
ClinicalTrials.gov Identifier:
NCT07378943
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
    Nov 2024 - Mar 2025
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Nusinersen and Risdiplam Cohorts:
    • Inclusion criteria
    • Patients with ≥1 SMA diagnosis (International Classification of Diseases, Tenth Revision, Clinical Modification \[ICD-10-CM\] codes: G12.0, G12.1, G12.9) at any time
    • Patients with ≥1 record of nusinersen or risdiplam in the data based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC) on or after December 23, 2016
    • Patients aged ≥ 2 at the index date
    • Patients with ≥ 1 quarter of clinical activities within 1 year prior to the index quarter (i.e., the quarter containing the index date)
    • Patients with ≥ 2 quarters of clinical activities in the first year of the follow-up
    • Exclusion criteria • None
    • Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:
    • Inclusion criteria
    • Patients with ≥1 SMA diagnosis (ICD-10-CM codes: G12.0, G12.1, G12.9) at any time
    • Patients with ≥1 record of OAV in the data based on relevant HCPCS codes and NDC on or after May 26, 2019
    • Patients under 2 years old at the index date
    • Patients with ≥ 2 quarters of clinical activities in their first year of follow-up
    • Exclusion criteria
    • • Treatment with nusinersen or risdiplam at any time

    Protocol 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.

    Trial Locations

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

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