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

NCT07321990 | Recruiting

• 1 other identifier: mSMAFRS-F
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

SMA

Keywords

SMA; Questionnaire; Translation; Validation

Outcome Measures

Primary Outcomes (1)

• Test-retest reliability

  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

Study Arms (1)

Treated patients

Other: Questionnaire and Physical Exam

Interventions

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.

Also known as: SMA-FRS

Treated patients

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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).

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Institut de Myologie, France: lead

Study Sites (1)

Association institut de Myologie

Paris, 75013, France

RECRUITING

MeSH Terms

Interventions

Surveys and Questionnaires; Restraint, Physical

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health; Behavior Control; Therapeutics; Immobilization

Central Study Contacts

Simone BIRNBAUM, PhD

CONTACT

+330142165879; s.birnbaum@institut-myologie.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2025
• First Posted: January 7, 2026
• Study Start: July 18, 2025
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: January 7, 2026

Record last verified: 2026-01

Locations

FR (1)