Clinical trial NCT05564936: The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis
Official title: The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis: a Validation Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Myasthenia Gravis
Lead sponsor: Ad scientiam
Sponsor class: OTHER
First posted: 2022-10-04
Enrollment: 144
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: 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: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: baseline
Secondary outcomes:
measure: To compare results between in-clinic digital tests and in-clinic standard tests, test to test
timeFrame: baseline
measure: To assess reproducibility between in-clinic digital tests and at-home digital tests
timeFrame: baseline, day 1, day 89, day 90
measure: To assess test-retest reliability of at-home digital tests
timeFrame: Day1, Day 2, Day 3, Day 87, Day 88, Day 89
measure: To compare results obtained with the at-home ME&MG composite score and the in-clinic standard QMG composite score
timeFrame: baseline, day 1
measure: To assess adverse events related to the use of the mobile application
timeFrame: through study completion, an average of 1 year
measure: To assess satisfaction and user experience with the smartphone application
timeFrame: through study completion, an average of 1 year
measure: To assess correlation of the at-home ME&MG composite score with the Myasthenia Gravis Activities of Daily Living (MG-ADL) score
timeFrame: baseline, day 90, day 365
measure: to assess daily activites
timeFrame: 12 months
measure: to assess depression
timeFrame: 12 months
measure: to assess pain
timeFrame: 12 months
measure: to assess insomnia
timeFrame: 12 months
measure: To assess at-home compliance to the ME&MG smartphone application
timeFrame: through study completion, an average of 1 year
measure: To assess quality of life
timeFrame: Baseline, day 90, Day 365
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado Denver
state: Colorado
city: Jacksonville
country: United States
facility: University of Florida Health
state: Florida
city: Bloomington
country: United States
facility: HealthParterns Institute
state: Indiana
city: Indianapolis
country: United States
facility: Indiana University Health
state: Indiana
city: Lexington
country: United States
facility: University of Kentucky
state: Kentucky
city: Lake Success
country: United States
facility: Neurological Associates of Long Island, P.C.
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Portland
country: United States
facility: OHSU
state: Oregon
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Knoxville
country: United States
facility: The University of Tennessee Medical Center
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt Health
state: Tennessee
city: Garches
country: France
facility: Hôpital Raymond Poincaré
city: Grenoble
country: France
facility: CHU Grenoble
city: Lille
country: France
facility: Hôpital Salengro
city: Nancy
country: France
facility: CHRU Nancy
city: Nantes
country: France
facility: CHU Nantes
city: Paris
country: France
facility: Pitié-Salpêtrière
city: Strasbourg
country: France
facility: CHU de Strasbourg - Hôpital de Hautepierre
city: Toulouse
country: France
facility: CHU Toulouse
Study officials:
References: