The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis: a Validation Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Ad scientiam
- 入组人数
- 144
- 试验地点
- 34
- 主要终点
- 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
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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
排除标准
- •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.
结局指标
主要结局
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
时间窗: baseline
Pearson correlation coefficient will be used to assess the relationship between digital tests versus standard tests
次要结局
- To compare results between in-clinic digital tests and in-clinic standard tests, test to test(baseline)
- to assess insomnia(12 months)
- To assess reproducibility between in-clinic digital tests and at-home digital tests(baseline, day 1, day 89, day 90)
- To assess test-retest reliability of at-home digital tests(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(baseline, day 1)
- To assess adverse events related to the use of the mobile application(through study completion, an average of 1 year)
- To assess satisfaction and user experience with the smartphone application(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(baseline, day 90, day 365)
- to assess depression(12 months)
- To assess quality of life(Baseline, day 90, Day 365)
- to assess daily activites(12 months)
- to assess pain(12 months)
- To assess at-home compliance to the ME&MG smartphone application(through study completion, an average of 1 year)