Holistic Assessment and Remote Digital MONitoring of mYasthenia Gravis Via MyoSense 360
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- Ad scientiam
- 入组人数
- 100
- 主要终点
- To explore the feasibility of using digital measures collected by MyoSense 360 to develop models to detect changes in disease trajectory in adults with generalized Myasthenia Gravis over a 12-month period, under real-life, unsupervised conditions.
研究概览
简要总结
The purpose of the HARMONY 360 investigation is to explore the feasibility of using MyoSense 360 digital measures to develop models capable of detecting clinically meaningful changes in generalized Myasthenia Gravis (gMG) trajectory over 12 months under real-world conditions. Its main objective is not yet to demonstrate the final conformity of the device (exploratory study under article 82), but to collect preliminary data of safety and clinical performance that will help to refine the final product and inform the subsequent pivotal study.
The primary objective focuses on developing models to detect disease trajectory changes defined by the clinically significant +/- 2 points MG-ADL threshold. Secondary objectives include assessing usability, adherence, and the feasibility of models for worsening/exacerbation prediction. Finally, risks and anticipated Adverse Device Effects (ADEs) associated with the device use will be monitored continuously throughout the investigation. Consistent with its Proof of Concept stage, the study defines no claims of clinical performance, effectiveness or safety.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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
- 否
入选标准
- •Age ≥ 18 years at screening
- •Patients with gMG diagnosis confirmed by the investigator based at least on one of the following:
- •Positive antibody testing for ACHR or MuSK antibodies; or,
- •LRP-4 antibodies or seronegative patients with additional positive single fiber examination; or,
- •Abnormal single-fiber EMG (SFEMG) jitter in at least one clinically weak muscle; or,
- •Objective evidence of improvement of clinical gMG symptoms following treatment with oral AChEI or positive pyridostigmine or neostigmine or edrophonium test
- •MGFA Class IIa to IVb
- •Having read the information sheet and signed the informed consent form
- •Owning and able to use a personal smartphone which software version is above 16 for iOS and 8 for Android included
- •Able to read languages in which the mobile application is available and able to understand pictograms (MyoSense 360 is available in local official languages)
- •Able to use an activity monitor (per Actigraph LEAP activity monitor wearability criteria; see vendor guidance)
- •For France only, affiliated with or benefiting from a social security system
排除标准
- •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.
- •Person under guardianship or curatorship
- •Current drugs or/and alcohol abuse that could influence performance on the tests, as judged by the investigator
- •Participant has participated in another interventional clinical study within 30 days prior to screening or who are currently enrolled in another study that, in the - Investigator's opinion, could interfere with their full participation in this study or confound the assessment of the subject or study outcomes
- •For France only, participant who has received more than €6,000 in compensation over the past 12 months for participating in human research, clinical trials, clinical investigations or performance studies.
研究组 & 干预措施
MyoSense 360
Performance of digital tests (only at D0), digital questionnaires and standard test in clinic at D0, D180 and D360 Use of MyoSense 360 at-home in between visits during 12 months of follow-up and answer ePRO
干预措施: MyoSense 360 (Device)
结局指标
主要结局
To explore the feasibility of using digital measures collected by MyoSense 360 to develop models to detect changes in disease trajectory in adults with generalized Myasthenia Gravis over a 12-month period, under real-life, unsupervised conditions.
时间窗: 12 months
The primary endpoint will be the ability of the model to detect changes categorized as a 3-class variable: * improved: decreased Myasthenia Gravis Activities of Daily Living (MG-ADL) total score ≥2 points * unchanged: increased or decreased MG-ADL total score \<2 points * worsening: increased MG-ADL total score by ≥2 points
次要结局
- To explore the feasibility of using digital measures collected by MyoSense 360 to develop models to predict sustained worsening within a one-month forward-looking window(1 month)
- To describe the association between MyoSense 360 active functional tests measures obtained at home without supervision and the standard reference Quantitative Myasthenia Gravis (QMG) sub-items performed under clinical supervision at baseline(Day 0 and Day 1)
- To describe the association between digital measures from MyoSense 360 (ActiGraph LEAP data collection, active smartphone functional tests and electronic Patient-Reported Outcomes) and clinical and patient-reported outcome measures(12 months)
- To describe the usability of MyoSense 360, including ActiGraph LEAP data collection, active functional tests and ePROs, from the patient's perspective.(Days 1, 180 and 345)
- To describe overall patient satisfaction and perceived benefits of using MyoSense 360 in daily life(Days 1, 180 and 345)
- To monitor the incidence, type, and severity of adverse events and device deficiencies related to the use of MyoSense 360.(390 days)
- To describe the association between MyoSense 360 active functional tests combined measures in a model, obtained at home, without supervision and the standard reference QMG sub-items scores performed under clinical supervision at baseline.(4 weeks)
- To describe participant compliance to the use of the MyoSense 360, including ActiGraph LEAP data collection all along the study, number of active functional tests performed and ePROs completed(12 months)