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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Myasthenia Gravis
• Intervention / Treatment: Device: ME&MG mobile application

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 144
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Pr Laforet; Phone Number: +33147107752; Email: pascal.laforet@aphp.fr

Study Locations

• France
  • Garches, France, 92380
    • Recruiting
    • Hôpital Raymond Poincaré
    • Contact: Pr Pascal Laforet; Phone Number: 01.47.10.77.36; Email: pascal.laforet@aphp.fr
    • Contact: Dr Edouard Berling
    • Contact: Dr Claire LEFEUVRE
    • Contact: Dr Alice ROUYER
    • Contact: Pr Guillaume NICOLAS
  • Grenoble, France
    • Not yet recruiting
    • CHU Grenoble
    • Contact: Dr Lagrange; Phone Number: +33 476767575; Email: elagrange@chu-grenoble.fr
    • Contact: Dr Lagrange
  • Lille, France
    • Recruiting
    • Hôpital Salengro
    • Contact: Dr Tard; Phone Number: +33 3 20 44 59 62; Email: CELINE.TARD@chru-lille.fr
    • Contact: Dr Tard
  • Nancy, France, 54035
    • Recruiting
    • CHRU Nancy
    • Contact: Dr Michaud; Phone Number: +33 3 83 85 85 85; Email: m.michaud@chru-nancy.fr
    • Contact: Dr Michaud
    • Contact: Pauline DUCATEL
  • Nantes, France
    • Recruiting
    • CHU Nantes
    • Contact: Dr Péréon; Phone Number: +33 0240083617; Email: Yann.Pereon@univ-nantes.fr
    • Contact: Dr Péréon
  • Paris, France, 75013
    • Recruiting
    • Pitié-Salpetrière
    • Contact: Anthony Behin, Dr; Phone Number: +33 142163774; Email: anthony.behin@aphp.fr
    • Contact: Anthony Behin, Dr
    • Contact: Tanya STOJKOVIC, Dr
  • Strasbourg, France, 67200
    • Completed
    • CHU de Strasbourg - Hôpital de Hautepierre
  • Toulouse, France
    • Recruiting
    • CHU Toulouse
    • Contact: Dr Cintas; Phone Number: +33 5 61 77 94 40; Email: cintas.p@chu-toulouse.fr
    • Contact: Dr Cintas
• United States
  • Colorado
    • Aurora, Colorado, United States, 80204
      • Recruiting
      • University of Colorado Denver
      • Contact: Thomas Ragole, Dr; Phone Number: 303-724-2188; Email: thomas.ragole@cuanschutz.edu
      • Contact: Dr Ragole
  • Florida
    • Jacksonville, Florida, United States, 32209
      • Recruiting
      • University of Florida Health
      • Contact: Michael T Pulley; Phone Number: (904) 383-1022; Email: michael.pulley@jax.ufl.edu
  • Indiana
    • Bloomington, Indiana, United States, 47401
      • Completed
      • HealthParterns Institute
    • Indianapolis, Indiana, United States, 46123
      • Recruiting
      • Indiana University Health
      • Contact: Sara M Takacs; Phone Number: 3179485450; Email: stakacs@iuhealth.org
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • Recruiting
      • University of Kentucky
      • Contact: Ima M Ebong; Phone Number: 8592571000; Email: Ima.Ebong@uky.edu
  • New York
    • Lake Success, New York, United States, 11042
      • Recruiting
      • Neurological Associates of Long Island, P.C.
      • Contact: Denis Ostrovskiy, MD; Phone Number: (516) 466-4700; Email: dostrovskiy@neuroli.com
  • North Carolina
    • Durham, North Carolina, United States, 27708
      • Recruiting
      • Duke University
      • Contact: Shruti Raja, Dr; Phone Number: 919-684-5422; Email: shruti.raja@duke.edu
      • Contact: Dr Raja
  • Oregon
    • Portland, Oregon, United States, 97239
      • Recruiting
      • OHSU
      • Contact: Nizar Chahin, Dr; Phone Number: 503-494-7772; Email: chahin@ohsu.edu
      • Contact: Dr Chahin
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19144
      • Recruiting
      • Thomas Jefferson University
      • Contact: Erika Wong, Dr; Phone Number: 215 955-4672; Email: Ericka.Wong@jefferson.edu
      • Contact: Dr Wong
  • Tennessee
    • Knoxville, Tennessee, United States, 37920
      • Not yet recruiting
      • The University of Tennessee Medical Center
      • Contact: Christopher Dr Scheiner; Phone Number: 856-521-6174; Email: cscheiner@utmck.edu
      • Contact: Dr Scheiner
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt Health
      • Contact: Lindsay Malatesta, Dr; Phone Number: 615 936-0600; Email: lindsay.malatesta@vumc.org
      • Contact: Dr Malatesta

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Aged over 18 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
• Subjects having initiated FcRn inhibitor therapy within the four weeks preceding 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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Healthy volunteers; Healthy volunteers will perform one in-clinic visit and will use the app at-home once	Device: ME&MG mobile application; 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.
Experimental: MG patients; MG patients will perform 3 in-clinic visits and use the ME&MG app at-home during 12 months	Device: ME&MG mobile application; 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.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Ad scientiam

Study record dates

Study Major Dates

• Study Start (Actual): January 24, 2024
• Primary Completion (Estimated): September 24, 2025
• Study Completion (Estimated): September 24, 2026

Study Registration Dates

• First Submitted: September 27, 2022
• First Submitted That Met QC Criteria: September 29, 2022
• First Posted (Actual): October 4, 2022

Study Record Updates

• Last Update Posted (Actual): April 11, 2025
• Last Update Submitted That Met QC Criteria: April 8, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Manifestations; Pathologic Processes; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: DOMYA

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No