NCT07474311Completed
A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
Real-World Evidence of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Analysis Based on REaDY Registry
Source: ClinicalTrials.gov · Last updated 20 Mar 2026
Brief summary (registry, in English)
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 213 (actual)
- First submitted
- 11 Mar 2026
- Start date
- 22 Nov 2011
- Primary completion date
- 13 Mar 2025
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07474311
What you commit to
- Study type: Observational
- Participants enrolled: 213
- From 22 Nov 2011 to the primary completion date, 13 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.
Other
- None.
Who the study is looking for
- Patients ≤18 years of age on the index date.
- Patients with genetically confirmed SMA.
- Patients with a known examination on the index date.
- Patients with a signed informed consent from their legal representative.
Age in the registry: - to 18 years · 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 | Prague | Czechia | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.