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临床试验/NCT07321990
NCT07321990已完成不适用

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

Institut de Myologie, France1 个研究点 分布在 1 个国家目标入组 60 人开始时间: 2025年7月18日最近更新:
干预措施

试验速览

阶段
不适用
状态
已完成
发起方
入组人数
60
试验地点
1
主要终点
Test-retest reliability

研究概览

简要总结

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.

详细描述

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.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
— 至 18 Years(Child, Adult)
性别
All
接受健康志愿者

入选标准

  • 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

排除标准

  • 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

研究组 & 干预措施

Treated patients

干预措施: Questionnaire and Physical Exam (Other)

结局指标

主要结局

Test-retest reliability

时间窗: 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).

次要结局

未报告次要终点

研究者

发起方
Institut de Myologie, France
申办方类型
Other
责任方
Sponsor

研究点 (1)

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