NCT07321990

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 60 (estimated)
• Sponsor: Institut de Myologie, France · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The modified SMAFRS is a patient-reported outcome measure (PROM) designed to assess the level of independence in ten activities of daily living (ADLs) within one's own environment for individuals with SMA. Activities include eating, dressing, bathing, toileting, grooming, turning in bed/adjusting bedclothes, transfers, walking, climbing stairs, and respiratory status. Each item is scored from 0 (fully dependent) to 5 (fully independent) by the patient or caregiver, maximum score of 40. The mSMAFRS has been used in several therapeutic and 9/21 2025-A00536-43\_PROTOCOLE\_V1.2\_20250707\_mSMAFRS-F observational studies. Feasibility, test-retest reliability, construct \& content validity has been demonstrated for the English version. However currently no French version exists. Therefore, each clinician or health professional translates the items themselves during consultation with patients. This has consequences for inter-rater reliability as translations are likely to differ from one clinician to another and for intra-rater reliability as translations may differ at each visit. Therefore, successive evaluations are compromised. It is essential that an official French version exists for both clinical and research use. The added benefit of the mSMAFRS to other functional scales (e.g., MFM) is its specificity in SMA and its simplicity to perform as it is patient-reported and not observer-rated. It is useful and relevant for both ambulant and non-ambulant individuals and it includes a respiratory item which is lacking in the other functional scales.

Conditions

• SMA

Interventions

Type	Name	Description
OTHER	Questionnaire and Physical Exam	Patients will answer the french mSMA-FRS during their introduction to treatment or treatment follow-up visit, then again, fifteen days after this visit.

Timeline

• Start date: 2025-07-18
• Primary completion: 2026-10-31
• Completion: 2026-10-31
• First posted: 2026-01-07
• Last updated: 2026-01-07

Locations

1 site across 1 country: France