CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain

NCT ID: NCT07231549

Last Updated: 2025-11-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-05-05

Study Completion Date

2026-12-31

Brief Summary

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The goal of this observational study is to set up a longitudinal data collection study to perform a long-term follow-up of SMA patients in a real-world setting, and to standardise and homogenize the data collection process for SMA patient's data.

Treated and untreated patients will be followed based on the routine clinical practice schedule.

Detailed Description

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This is a retrospective, prospective, multicentre, non-randomized data collection study in Spain. The dataset collected is based on the TREAT-NMD SMA Core Dataset and the CuidAME steering committee consensus. The study collects pseudo-anonymized data, including data on genetic test results, clinical diagnoses, treatment, and outcome measures.

Patients will be followed based on the routine clinical practice schedule. Motor outcomes will be captured regularly, following the SMA standards of for care, for patients with a DMT every 6 months or at least once per year in patients without DMTs, for at least a 5-year follow-up period initially, which could be extended under agreement with funders of the study.

Participation in this study does not modify or influence any clinical decisions regarding the treatment the patient may receive, clinical assessments performed or frequency of visits.

The study will not collect data from patients participating in a clinical trial from the time they are enrolled. Data collected before the enrolment may be used, and data collection may continue after the end of the clinical trial. Data generated during a clinical trial could be used if written permission from the clinical trial sponsor is obtained.

Conditions

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Spinal Muscular Atrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Subjects with Spinal Muscular Atrophy 5q

Subjects with Spinal Muscular Atrophy 5q

No interventions assigned to this group

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fundació Sant Joan de Déu

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Andrés Nascimento

Role: STUDY_CHAIR

Hospital Sant Joan de Deu

Mónica Povedano Panadés

Role: STUDY_CHAIR

Hospital Universitari de Bellvitge

Francina Munell Casadesús

Role: STUDY_CHAIR

Hospital Universitari Vall d'Hebrón

Miguel Angel Fernández García Miguel Angel Fernández García

Role: STUDY_CHAIR

Hospital Universitario La Paz

Inmaculada Pitarch Castellano

Role: STUDY_CHAIR

Hospital Universitario La Fe

Juan Francisco Vázquez Costa

Role: STUDY_CHAIR

Hospital Universitario La Fe

Maria Grazia Cattinari

Role: STUDY_CHAIR

Fundación Atrofia Muscular Espinal

Rocío Calvo

Role: STUDY_CHAIR

Hospital Universitario Regional de Málaga

Eduardo Martínez Salcedo

Role: STUDY_CHAIR

Hospital Universitario Virgen de la Arrixaca

Antonio Moreno Escribano

Role: STUDY_CHAIR

Hospital Universitario Virgen de la Arrixaca

Julita Medina Cantillo

Role: STUDY_CHAIR

Hospital Sant Joan de Deu

Locations

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Hospital Sant Joan de Déu

Esplugues de Llobregat, , Spain

Site Status RECRUITING

Countries

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Spain

Central Contacts

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CUIDAME Project Manager

Role: CONTACT

0034 936 009 751

Related Links

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Other Identifiers

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EOM-24-02

Identifier Type: -

Identifier Source: org_study_id

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