NCT07403214Completed
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data
Source: ClinicalTrials.gov · Last updated 11 Feb 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 4805 (actual)
- First submitted
- 4 Feb 2026
- Start date
- 4 Jun 2025
- Primary completion date
- 30 Jun 2025
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07403214
What you commit to
- Study type: Observational
- Participants enrolled: 4805
- From 4 Jun 2025 to the primary completion date, 30 Jun 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.
Other
- Patients with SMA type 4.
- Patients with claims of multiple DMTs in their birth year.
Who the study is looking for
- 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.
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 |
This page is information, not medical advice. Discuss any trial with your doctor.