Reproducibility of the French mSMAFRS questionnaire
Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
This study will test whether a French translation of the mSMAFRS questionnaire gives consistent answers when adults with SMA complete it at a clinic visit and again fifteen days later.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 60 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Institut de Myologie, France Academic / other |
| Locations | 1 site (Paris) |
| Trial ID | NCT07321990 on ClinicalTrials.gov |
What this trial studies
The study uses the patient-reported modified SMA Functional Rating Scale (mSMAFRS) translated into French and administered to adult patients with genetically confirmed SMA during routine follow-up. Participants complete the mSMAFRS-F at the visit and then again 15 days later to measure test-retest reliability using statistics such as the intraclass correlation coefficient (ICC). The protocol also examines how mSMAFRS-F scores relate to other outcome measures to check construct validity. The goal is to produce an official, stable French version to avoid ad hoc clinician translations that compromise consistency.
Who should consider this trial
Good fit: Adults (≥18) with genetically confirmed SMA who speak French, can complete questionnaires without significant cognitive impairment, and can comply with study procedures are ideal candidates.
Not a fit: People who do not speak French, have significant cognitive impairment preventing questionnaire completion, are under legal protection measures, or cannot comply with protocol requirements are unlikely to benefit from participation.
Why it matters
Potential benefit: If successful, an official French mSMAFRS would give clinicians and researchers a reliable, patient-reported tool to track daily function in French-speaking adults with SMA.
How similar studies have performed: The English mSMAFRS has demonstrated feasibility, test-retest reliability, and validity in prior work, but an official French version has not yet been validated.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Paris
- Association institut de Myologie — Paris, France (Recruiting)
Study contacts
- Study coordinator: Simone BIRNBAUM, PhD
- Email: s.birnbaum@institut-myologie.org
- Phone: +330142165879
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.