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

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.

Completed

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Key information

Conditions

SMA

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Association institut de Myologie

Paris, 75013, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

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

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

Treatment and study plan

Questionnaire and Physical Exam

Other

Patients will answer the french mSMA-FRS during their introduction to treatment or treatment follow-up visit, then again, fifteen days after this visit.

Other names: SMA-FRS

Primary outcomes

Test-retest reliability

Time frame: up to 2 weeks

ICC is a good estimate of test-retest reliability. It is expected that mSMAFRS-F is highly reliable (ICC>0.9).

Sponsors and collaborators

Lead sponsor

Institut de Myologie, France

Other

Registry information

Acronym: mSMAFRS-F

Important dates

Study start: 2025
Primary completion: 2026
Study completion: 2026
First posted: Jan 7, 2026
Registry last updated: Jun 17, 2026

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.

Published trials that share one or more normalized conditions with this study.

Completed