The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis

Study Overview
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Brief Summary
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ME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG. ME&MG contains digital active tests for the assessment of ptosis, breathing, dysarthria, upper- and lower-limb (arms and legs) weakness, treatment follow-up, and validated e-questionnaires related to daily activities, pain, fatigue, sleep, and depression disorders. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone with ME&MG versus the standard in-clinic testing, as well as to evaluate the safety of the solution, its usability and satisfaction.

Detailed Description
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Myasthenia gravis (MG) is an autoimmune neuromuscular disease characterised by fatigable muscle weakness due to autoantibodies targeting components of the neuromuscular junction. Deficits are measured using validated clinical scales such as the Quantitative Myasthenia Gravis score (QMG) QMG, Myasthenia Gravis Composite Score (MCS), Myasthenia Gravis Activities of Daily Living score (MG-ADL), etc., Unfortunately, this type of assessment might require expensive material, time, and personnel training, which make MG assessment difficult for clinicians in routine care. Research on the matter has highlighted the importance of Patient-Reported Outcomes (PROs), composite measures of disease severity and quality-of-life measurements. This has led to the development of a number of tools for the self-assessment of PROs with validated questionnaires. However, there is no tool that overcomes the constraints of standard tests such as the QMG while allowing for the collection of objective data and PROs between visits. ME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG. ME&MG contains digital active tests for the assessment of ptosis, breathing, dysarthria, upper- and lower-limb (arms and legs) weakness, treatment follow-up, and validated e-questionnaires related to daily activities, pain, fatigue, sleep, and depression disorders. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone with ME&MG versus the standard in-clinic testing, as well as to evaluate the safety of the solution, its usability and satisfaction.

Official Title
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The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis: a Validation Study

Conditions
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Myasthenia Gravis

Intervention / Treatment
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* Device: ME&MG mobile application


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 Years to 60 years Diagnosed with gMG at screening with generalized muscle weakness meeting the clinical criteria for diagnosis of MG, as defined by the MGFA Clinical Classification Class II, III, or IV, and likely not in need of a respirator for the duration of the study, as judged by the investigator With positive serologic testing for anti-AChR autoantibody at screening Have read the information sheet and signed the informed consent form Own a personal smartphone which software version is above 13 for IOS and 8 for Android included Able to use a smartphone Able to read language in which the mobile application is available (French, English, Spanish) and able to understand pictograms Exclusion Criteria: Subjects having undergone thymectomy Plasma Exchange Therapy or Intravenous Immunoglobulin Therapy within four weeks of screening Known medical or psychological condition(s) or risk factor(s) that, as judged by the investigator, might interfere with the subject's full participation in the study, pose any additional risk for the subject, or confound the assessment of the subject or outcome of the study. Pregnant and nursing women Person under guardianship or curatorship Current drugs or/and alcohol abuse that could influence performance on the tests, as judged by the investigator Participant included in another ME&MG clinical study Participant has participated in another clinical study within the previous 30 days of screening or is currently participating in another study that, in the opinion of the Investigator, might interfere with the subject's full participation in the study or confound the assessment of the subject or outcome of the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MG patients<br>MG patients will perform 3 in-clinic visits and use the ME&MG app at-home during 12 months | Device: ME&MG mobile application<br>* ME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG.<br>|
| Other: Healthy volunteers<br>Healthy volunteers will perform one in-clinic visit and will use the app at-home once | Device: ME&MG mobile application<br>* ME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To demonstrate that ME&MG tests results performed at-home, under unsupervised condition, are correlated to standard tests results performed in-clinic, under supervised conditions | Pearson correlation coefficient will be used to assess the relationship between digital tests versus standard tests | baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To compare results between in-clinic digital tests and in-clinic standard tests, test to test | pearson correlation coefficient between digital tests and standard tests | baseline | 
| To assess reproducibility between in-clinic digital tests and at-home digital tests | intraclass correlation coefficient | baseline, day 1, day 89, day 90 | 
| To assess test-retest reliability of at-home digital tests | intraclass correlation coefficient mean k raters (ICCk; k=day) | Day1, Day 2, Day 3, Day 87, Day 88, Day 89 | 
| To compare results obtained with the at-home ME&MG composite score and the in-clinic standard QMG composite score | Pearson correlation coefficient between digital composite score and QMG score | baseline, day 1 | 
| To assess adverse events related to the use of the mobile application | Descriptive analysis of adverse events (AEs) related to the use of the application | through study completion, an average of 1 year | 
| To assess satisfaction and user experience with the smartphone application | descriptive analysis of questionnaires resulsts | through study completion, an average of 1 year | 
| To assess correlation of the at-home ME&MG composite score with the Myasthenia Gravis Activities of Daily Living (MG-ADL) score | Pearson's correlation coefficient between ME&MG composite score and MG-ADL scores | baseline, day 90, day 365 | 
| to assess daily activites | Myasthenia Gravis Activities of Daily Living (MG-ADL) | 12 months | 
| to assess depression | Patient Health Questionnaire-8 (PHQ8) | 12 months | 
| to assess pain | pain likert scale | 12 months | 
| to assess insomnia | Insomnia Severity Index | 12 months | 
| To assess at-home compliance to the ME&MG smartphone application | descriptive analysis of adherence data | through study completion, an average of 1 year | 
| To assess quality of life | 36-Item Short Form Survey (SF-36) | Baseline, day 90, Day 365 |