RecruitingOBSERVATIONAL

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

This research is looking at a French version of a special questionnaire called the mSMAFRS. This questionnaire helps adults with Spinal Muscular Atrophy (SMA) describe how well they can do everyday tasks, such as eating, dressing, and moving around. The main goal is to make sure this French version gives consistent results over time. This is important because it will help doctors and researchers accurately understand how SMA affects patients in France and track any changes. Currently, there isn't an official French version, so having one will help everyone use the same standard. Participants will simply answer the questionnaire during two separate visits, about fifteen days apart, as part of their regular check-ups.

At a glance

Status

Recruiting

Sponsor

Institut de Myologie, France

Enrolment target

Start

18 Jul 2025

Estimated completion

31 Oct 2026

SMA

What is this study about?

Imagine filling out a questionnaire about your daily activities, like how easily you can eat or get dressed. Now, imagine doing it again a couple of weeks later. If your answers are pretty much the same, it means the questionnaire is reliable and gives a good picture of your abilities. This study is doing exactly that for a specific questionnaire called the mSMAFRS, but in French, for adults living with Spinal Muscular Atrophy (SMA).

The mSMAFRS questionnaire asks about ten simple daily activities, from eating and dressing to walking and breathing. Each activity is given a score from 0 (meaning you need full help) to 5 (meaning you can do it completely on your own). The highest possible total score is 40. This tool is really helpful because it focuses on what matters most to individuals – their independence in their own home.

Right now, there isn't an official French version of this important questionnaire. This means that different doctors might translate the questions slightly differently, which can make it hard to compare results over time or between different patients. This study aims to create a clear, standardised French version, ensuring everyone uses the same wording. This consistency is vital for both everyday patient care and for future research into SMA, helping to ensure that assessments are accurate and that potential new treatments can be properly evaluated.

Key takeaways

This study helps make an important SMA questionnaire available in French.
It checks if the French questionnaire gives consistent results over time.
You'll answer questions about daily activities, not take new medicines.
Participation involves two simple questionnaire sessions during routine visits.
This research aims to improve how SMA is assessed for French-speaking patients.

Who may be eligible?

To be part of this study, you need to be at least 18 years old and have a confirmed diagnosis of Spinal Muscular Atrophy (SMA) through genetic testing. It's also important that you can understand and speak French well enough to complete the questionnaire, and that you can follow all the study instructions.

You won't be able to join if you have a legal guardian or if you struggle to understand or speak French. Similarly, if you have any memory or thinking problems that would make it hard to answer the questionnaire accurately, or any other health or social issues that your doctor thinks would interfere with the study, you wouldn't be included.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a genetically confirmed diagnosis of SMA?
Can you speak and understand French well?
Are you generally able to follow instructions without significant memory problems?

Answer every question to see your result.

What does participation involve?

If you decide to take part, your involvement will be quite straightforward. You'll complete the French version of the mSMAFRS questionnaire, which asks about your daily activities, during two of your regular clinic visits. These two visits will be about fifteen days apart. You won't need to take any new medication or have any extra tests beyond what you already have for your routine care. The total time you'll be involved with the study will not extend beyond these two questionnaire completions during your follow-up appointments.

Potential risks and benefits

There are no significant physical risks involved in this study as it only involves answering a questionnaire. Completing the questionnaire twice might take a little extra time during your clinic visits. The main benefit is that you'll be helping to create a reliable French version of an important tool for people with SMA, which will ultimately improve care and research for others. You are free to withdraw from the study at any time without giving a reason, and it will not affect your usual medical care.

Locations (1)

Association institut de Myologie
Verified postcode
Paris, France· Recruiting

Common questions

What is SMAFRS?

SMAFRS is a questionnaire that helps measure how well people with Spinal Muscular Atrophy (SMA) can perform everyday tasks like eating, dressing, and walking.

Why do you need a French version?

Currently, doctors in France might translate the questionnaire differently, which makes it hard to compare results. An official French version will ensure everyone uses the same clear questions.

What will I have to do if I join?

You will fill out the French SMAFRS questionnaire twice, about 15 days apart, during your regular clinic visits.

Will I get any new medicine or treatment?

No, this study only involves answering questionnaires. You won't receive any new medications or treatments through this research.

Are there any side effects?

No, as this study only involves questionnaires and no treatments, there are no physical side effects or risks.

How to find out more

Simone BIRNBAUM, PhD

s.birnbaum@institut-myologie.org +330142165879 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.