SMAmSMAFRS-FNCT07321990
Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
An observational study of SMA, run by Institut de Myologie, France at 1 site in France.
Trial 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… Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- SMA
- What is being tested?
- Questionnaire and Physical Exam
- How many people
- 60 participants
- Where
- 1 siteFrance
- Study dates
- 18 Jul 2025to 8 Apr 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: This study will be conducted in 60 adult's patients with SMA ≥18 years (ambulatory or not, treatment naïve or not).
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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
- Being tested
- Questionnaire and Physical Exam
- Keywords
- SMA, Questionnaire, Translation, Validation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Questionnaire and Physical ExamOther
Also called SMA-FRS
Patients will answer the french mSMA-FRS during their introduction to treatment or treatment follow-up visit, then again, fifteen days after this visit.
| Study group | Role | What they receive |
|---|---|---|
| Treated patients | Not described Receives: Other: Questionnaire and Physical Exam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Test-retest reliability
ICC is a good estimate of test-retest reliability. It is expected that mSMAFRS-F is highly reliable (ICC>0.9).
Measured: up to 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2026
- Study start18 Jul 2025
- Primary completion8 Apr 2026
- Study completion8 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SMAStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07321990
- Sponsor's protocol ID
- mSMAFRS-F
- Acronym
- mSMAFRS-F
- Status last verified
- Jun 2026
- First submitted
- 12 Dec 2025
- Last updated
- 17 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
- ClinicalTrials.gov ID
- NCT07321990
- Acronym / sponsor ID
- mSMAFRS-F / mSMAFRS-F
- Conditions
- SMA
- Keywords
- SMA, Questionnaire, Translation, Validation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Study start
- 18 Jul 2025 (actual)
- Primary completion
- 8 Apr 2026 (actual)
- Study completion
- 8 Apr 2026 (actual)
- First posted
- 7 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treated patients | – | Other: Questionnaire and Physical Exam |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- This study will be conducted in 60 adult's patients with SMA ≥18 years (ambulatory or not, treatment naïve or not).
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Questionnaire and Physical ExamAlso: SMA-FRSPatients will answer the french mSMA-FRS during their introduction to treatment or treatment follow-up visit, then again, fifteen days after this visit. | Other | Treated patients |
Primary outcome
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).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of SMA, run by Institut de Myologie, France at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Test-retest reliability (time frame: up to 2 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jun 2026.