Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules

Study Overview
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Brief Summary
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Neuromuscular diseases include more than 200 rare disorders affecting muscles or the nervous system. Functional scales assessing motor function are tools allowing to measure the evolution of motor impairment of patients with a neuromuscular disease. They are frequently used as main outcome measures in clinical trials which are currently in full development thanks to advances in genetic and drug researches. Among the available scales, the Motor Function Measure (MFM), a scale consisting of 32 items, had shown good metrological properties in terms of validity, reliability and sensitivity to change, regardless of the diagnosis and extent of motor impairment. By exploring the potential of digital technologies applied to MFM, investigators created a completion module composed of animations with different playful and informative scenarios displayed on a digital tablet. The main purpose of this project is to conduct a multicentre study to assess reliabilities of the MFM completed using the MFM-Play. Expected benefits of this project are to improve the experience and the participation of the children to who is often imposed multiple assessments during the numerous follow-up visits of clinical trials, while maintaining good metrological properties of the MFM.

Official Title
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Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules

Conditions
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Neuromuscular Diseases

Intervention / Treatment
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* Other: Items completion with or without a MFM-Play


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a neuromuscular disease confirmed by the clinic, electroneuromyography (ENMG), biology, muscular biopsy, or genetic aged ≥ 2 yo and ≤ 60 yo having been informed and not objecting to participating in the study (information and non-opposition of parents/holders of parental authority for minor patients) Exclusion Criteria: participating in other interventional research with an exclusion period that is still ongoing or that may interfere with the results of this study having had surgery or illness impacting their motor function within the last 6 months having understanding and/or behavioral deficiencies preventing rigorous assessment Patient placed under legal protection (this includes guardianship, curatorship and safeguard of justice)

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| MFM-Play<br>Neuromuscular disease patients completed MFM using MFM-Play | Other: Items completion with or without a MFM-Play<br>* Patients will completed 3 MFM during the follow-up, two times using the MFM-Play, and one without the MFM-Play Delay between each MFM completion will be 1 month ± 15 days<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Comparison of item scoring between 2 MFM completion. | Level of agreement between MFM item scores reported by physiotherapists during 2 different MFM completion using the MFM-Play. Level of agreement between MFM item scores reported by a same physiotherapist during a MFM completion using the MFM-Play and a MFM completed conventionally. | 3 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Motor Function Assessment, Reliability study, Patient Participation