Study of the Reproducibility of the French Version of the Modified SMAFRS Scale (mSMAFRS-F)

January 5, 2026 updated by: Institut de Myologie, France

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

This study will be conducted in 60 adult's patients with SMA ≥18 years (ambulatory or not, treatment naïve or not).

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Treated patients
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.
Other Names:
  • SMA-FRS

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
up to 2 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institut de Myologie, France

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 18, 2025

Primary Completion (Estimated)

October 31, 2026

Study Completion (Estimated)

October 31, 2026

Study Registration Dates

First Submitted

December 12, 2025

First Submitted That Met QC Criteria

January 5, 2026

First Posted (Actual)

January 7, 2026

Study Record Updates

Last Update Posted (Actual)

January 7, 2026

Last Update Submitted That Met QC Criteria

January 5, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • mSMAFRS-F

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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