A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
Completed
Conditions studied: Muscular Atrophy, Spinal
In brief
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.
Key facts
- Study ID
- NCT07378943
- Run by
- Novartis Pharmaceuticals
- People needed
- 4114
- Starts
- 2024-11-06
- Expected to finish
- 2025-03-30
- Last updated by the study team
- 2026-01-30
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Treatment with nusinersen or risdiplam at any time
Where it is running
- Novartis — East Hanover, New Jersey, United States
Full record on ClinicalTrials.gov
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