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NCT Number: NCT07231549

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

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.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Sant Joan de Déu

Esplugues de Llobregat, Spain

Location status: Recruiting

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Motor and functional status (WHO)

    Time frame: Yearly through study completion, an average of 5 years

    Motor status will be captured (all patients): WHO motor milestones: checklist that assesses whether a child can sit independently, crawl, stand with/without support, and walk with/without support. Changes in this score (loss or gain in function) and maintenance of score throughout study duration is assessed.

  2. Motor and functional status (CHOP INTEND)

    Time frame: Yearly through study completion, an average of 5 years

    Motor status will be captured (patients below two years of age): Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): maximum score of 64.

  3. Motor and functional status (HFMSE)

    Time frame: Yearly through study completion, an average of 5 years

    Motor status will be captured (patients over 36 months of age): Hammersmith Functional Motor Scale Expanded (HFMSE): maximum score of 66.

  4. Motor and functional status (RULM)

    Time frame: Yearly through study completion, an average of 5 years

    Motor status will be captured (patients obver 36 months of age): Revised Upper Limb Module (RULM): maximum score of 37.

  5. Motor and functional status (6MWT)

    Time frame: Yearly through study completion, an average of 5 years

    Motor status will be captured (ambulant patients): 6-Minute-Walk Test (6MWT): walked distance within 6 min.

Secondary outcomes

  1. Clinical SMA characteristics: mechanical ventilation

    Time frame: Through study completion, an average of 5 years

    Change in mechanical ventilation prevalence: percent of participants who required ventilatory support (invasive or non-invasive)

  2. Clinical SMA characteristics: external nutrition

    Time frame: Yearly through study completion, an average of 5 years

    Change in external nutrition prevalence: percent of participants who required external nutrition support

  3. Clinical SMA characteristics: scoliosis

    Time frame: Yearly through study completion, an average of 5 years

    Change in scoliosis presence or scoliosis surgery prevalence: percent of participants who presented scoliosis and percent of participants with spinal surgery.

  4. Clinical SMA characteristics: neurodevelopmental developoment

    Time frame: Yearly through study completion, an average of 5 years

    Prevalence of neurodevelopmental disorders: percentage of patients who, after assessment by a neuropaediatrician, present with language, behavior, social or understanding alterations

  5. Clinical SMA characteristics: treatment sequence

    Time frame: Yearly through study completion, an average of 5 years

    change in treatments: distribution of patients in the different possible treatment sequences

Study contacts

Contact information is provided by the study sponsor or research team.

CUIDAME Project Manager

CONTACT

[email protected]

0034 936 009 751

Sponsors and collaborators

Lead sponsor

Fundació Sant Joan de Déu

Other

Registry information

Acronym: CUIDAME

Important dates

Study start
2020
Primary completion
2026
Study completion
2026
First posted
Nov 17, 2025
Registry last updated
Nov 17, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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