Actively Recruiting
CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
Led by Fundació Sant Joan de Déu · Updated on 2025-11-17
1000
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to collect long-term data from patients with Spinal Muscular Atrophy (SMA) in Spain to better understand the disease in a real-world setting. It is an observational, multicenter, non-randomized study that follows both treated and untreated patients according to routine clinical practice. The study seeks to standardize and unify the data collection process for SMA patients, focusing on motor and functional outcomes over time. Patients with SMA will be followed regularly based on standard care guidelines. Those receiving disease-modifying treatments (DMTs) will have motor outcomes recorded every six months, while untreated patients will be assessed at least once a year. The initial follow-up period is planned for at least five years, with possible extension depending on study funding. The study collects pseudo-anonymized data including genetic test results, clinical diagnoses, treatment history, and various outcome measures without affecting clinical care decisions. Participants' motor and functional status will be evaluated yearly using several standardized scales such as WHO, CHOP INTEND, HFMSE, RULM, and the 6-minute walk test. Additional clinical features like mechanical ventilation use, nutrition support, scoliosis, neurodevelopment, and treatment sequences will also be monitored annually. Data collection aligns with routine clinical visits, and participation does not change treatment or visit schedules. The study excludes data collected during any clinical trial unless permission is granted, and data may continue to be collected after clinical trial participation ends.
CONDITIONS
Brief Title
CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed 5q Spinal Muscular Atrophy
- Written informed consent obtained from patient or caregiver (except for deceased pediatric patients)
You will not qualify if you...
- Diagnosis of SMA types other than 5q SMA
- Patient lacks legal capacity to understand the nature and consequences of the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 5 years, possibly extended
Participants who undergo routine care are observed. Motor and functional status assessments and clinical characteristics are collected regularly following standard care schedules.
Visits every 6 months for participants with disease-modifying treatments or at least once per year for those without
Trial Site Locations
Total: 1 location
1
Hospital Sant Joan de Déu
Esplugues de Llobregat, Spain
Actively Recruiting
Research Team
C
CUIDAME Project Manager
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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