CuidAME: Spain's longitudinal registry for spinal muscular atrophy
CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
This project collects long-term clinical and treatment data from children and adults with genetically confirmed 5q spinal muscular atrophy in Spain to track outcomes over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1000 (estimated) |
| Sex | All |
| Sponsor | Fundació Sant Joan de Déu Academic / other |
| Locations | 1 site (Esplugues de Llobregat) |
| Trial ID | NCT07231549 on ClinicalTrials.gov |
What this trial studies
CuidAME is a retrospective and prospective multicenter registry in Spain that collects pseudo-anonymized clinical, genetic, treatment, and outcome data using the TREAT‑NMD SMA Core Dataset plus steering committee additions. Patients with genetically confirmed 5q SMA, treated or untreated, are followed according to routine clinical practice, with motor outcomes captured every six months for those on disease-modifying therapies and at least annually for those not on DMTs. Data are collected for an initial minimum of five years and may be extended with funder agreement, and participation does not change clinical care or visit schedules. Data from concurrent clinical trials are not collected during trial enrollment unless sponsor permission is obtained.
Who should consider this trial
Good fit: Ideal candidates are children and adults in Spain with genetically confirmed 5q SMA who (or whose caregivers) can provide informed consent and receive care at participating centers.
Not a fit: Patients with non-5q SMA, those unable to provide consent without a legal representative, and those actively enrolled in clinical trials (while they remain enrolled) may not benefit from participation.
Why it matters
Potential benefit: If successful, the registry could improve understanding of real-world disease progression and treatment effects in Spain, guiding clinical care and future research.
How similar studies have performed: Other national and international SMA registries based on TREAT‑NMD principles have successfully described natural history and real-world treatment outcomes, so this approach aligns with established methods.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Genetically confirmed 5q SMA * Patient or caregivers written informed consent has been obtained (except for paediatric patients deceased). Exclusion Criteria: * Further types of SMA (non-5q SMA). * Patient without legal capacity who cannot understand the nature, significance, and consequences of the project.
Where this trial is running
Esplugues de Llobregat
- Hospital Sant Joan de Déu — Esplugues de Llobregat, Spain (Recruiting)
Study contacts
- Study coordinator: CUIDAME Project Manager
- Email: info@registro-cuidame.org
- Phone: 0034 936 009 751
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.