NCT07378943Completed
A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy (SMA) Receiving Disease Modifying Therapies (DMT) - Real-world Analysis Using the Komodo Claims Data
Source: ClinicalTrials.gov · Last updated 30 Jan 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 4114 (actual)
- First submitted
- 23 Jan 2026
- Start date
- 6 Nov 2024
- Primary completion date
- 30 Mar 2025
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07378943
What you commit to
- Study type: Observational
- Participants enrolled: 4114
- From 6 Nov 2024 to the primary completion date, 30 Mar 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Treatment with nusinersen or risdiplam at any time
Other
- Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:
Who the study is looking for
- 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
- 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
Criteria the registry did not label
- Nusinersen and Risdiplam Cohorts:
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Novartis Coordinator of this site? Confirm its status | East Hanover | United States | Unknown status |
Related publications
This page is information, not medical advice. Discuss any trial with your doctor.