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

Myasthenia Gravis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05564936
• Other study ID #: DOMYA
• Status: Recruiting
• Phase: N/A
• Start date: January 24, 2024
• Est. completion date: September 15, 2025

Study information

• Verified date: April 2024
• Source: Ad scientiam
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 144
• Est. completion date: September 15, 2025
• Est. primary completion date: March 15, 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis

Intervention

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.

Locations

Country	Name	City	State
France	Hôpital Raymond Poincaré	Garches
France	CHRU Nancy	Nancy
France	CHU de Strasbourg - Hôpital de Hautepierre	Strasbourg
United States	Indiana University Health	Indianapolis	Indiana
United States	University of Florida Health	Jacksonville	Florida
United States	Neurological Associates of Long Island, P.C.	Lake Success	New York
United States	University of Kentucky	Lexington	Kentucky

Sponsors (1)

Lead Sponsor	Collaborator
Ad scientiam

Countries where clinical trial is conducted

United States,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	To assess reproducibility between in-clinic digital tests and at-home digital tests	intraclass correlation coefficient	baseline, day 1, day 89, day 90
Secondary	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
Secondary	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
Secondary	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
Secondary	To assess satisfaction and user experience with the smartphone application	descriptive analysis of questionnaires resulsts	through study completion, an average of 1 year
Secondary	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
Secondary	to assess daily activites	Myasthenia Gravis Activities of Daily Living (MG-ADL)	12 months
Secondary	to assess depression	Patient Health Questionnaire-8 (PHQ8)	12 months
Secondary	to assess pain	pain likert scale	12 months
Secondary	to assess insomnia	Insomnia Severity Index	12 months
Secondary	To assess at-home compliance to the ME&MG smartphone application	descriptive analysis of adherence data	through study completion, an average of 1 year
Secondary	To assess quality of life	36-Item Short Form Survey (SF-36)	Baseline, day 90, Day 365