CompletedNot Applicable

Study of the Reproducibility of the French Version of the Modified SMAFRS Scale

France60 participantsStarted 2025-07-18

Plain-language summary

The goal of this observational study is to evaluate the test-retest reliability of the French version of the mSMAFRS in adult patients with SMA. The main question\[s\] is to answer if: * ICC is a good estimate of test-retest reliability? * mSMAFRS-F correlate with other outcome measures? Participants will simply answer a french version of the SMA-FRS questionnaire during their routine follow-up visit then again fifteen days later.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Age ≥ 18 years * Genetically confirmed SMA * Able to comply with all protocol requirements (no significant cognitive impairment) * French-speaking * Affiliated or beneficiary of a social security scheme Exclusion Criteria: * Persons subject to a legal protection measure * Subjects unable to complete the French version of the questionnaire mSMAFRS-Fbecause of lack of French fluency * Subjects unable to complete the French version of the questionnaire mSMAFRS-Fbecause of cognitive impairment * Inability to comply with protocol requirements * Any medical and social conditions that could interfere with the study under theappreciation of the medical coordinator

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Test-retest reliability

Timeframe: up to 2 weeks

Trial details

NCT IDNCT07321990

SponsorInstitut de Myologie, France

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2026-04-08

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