Actively Recruiting
Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
Led by Institut de Myologie, France · Updated on 2026-01-07
60
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the test-retest reliability of the French version of the modified SMA Functional Rating Scale (mSMAFRS) in adult patients with spinal muscular atrophy (SMA). The study investigates whether the Intraclass Correlation Coefficient (ICC) is a good measure of reliability and if the mSMAFRS-F correlates well with other outcome measures. The goal is to establish an official French version of this patient-reported scale for clinical and research use, as the current lack of a standardized French version affects consistency in assessments. Participants will complete the French mSMAFRS questionnaire during their routine treatment introduction or follow-up visit and then again fifteen days later. This questionnaire assesses independence in ten daily activities such as eating, dressing, bathing, toileting, grooming, mobility, and respiratory status. The study focuses on collecting responses at these two time points to determine the reproducibility of the scale. During the study, participants will answer the questionnaire twice within a two-week interval as part of their usual care visits. Researchers will analyze the consistency of the responses over this period to assess test-retest reliability. The primary outcome is the reliability of the questionnaire results up to two weeks apart. The study involves adults with genetically confirmed SMA who speak French and can comply with the protocol, with total participation lasting about two weeks between assessments.
CONDITIONS
Brief Title
Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age �3A 18 years
- Genetically confirmed spinal muscular atrophy (SMA)
- Able to comply with all protocol requirements (no significant cognitive impairment)
- French-speaking
- Affiliated or beneficiary of a social security scheme
You will not qualify if you...
- Persons subject to a legal protection measure
- Unable to complete the French mSMAFRS questionnaire due to lack of French fluency
- Unable to complete the French mSMAFRS questionnaire due to cognitive impairment
- Inability to comply with protocol requirements
- Any medical or social conditions that could interfere with the study as determined by the medical coordinator
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.
Duration - Up to 2 weeks
Participants complete the French mSMA-FRS questionnaire during a treatment introduction or follow-up visit, then again 15 days later to assess test-retest reliability.
2 visits (in-person)
Trial Site Locations
Total: 1 location
1
Association institut de Myologie
Paris, France, 75013
Actively Recruiting
Research Team
S
Simone BIRNBAUM, PhD
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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