Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- MGFA-PIS: Myasthenia Gravis Post intervention status
研究概览
简要总结
This observational study seeks to investigate the underlying processes of myasthenia gravis by employing multimodal monitoring techniques. By integrating digital biomarkers alongside clinical monitoring, we aim to enhance the detection of disease activity and establish correlations between digital measures, clinical scores and various questionnaires including sores on quality of life, sleep quality or activities of daily living. Primarily including patients treated with newly approved drugs, it aims at improving and monitoring the efficacy and safety of treatment and allowing a more individualized treatment.
详细描述
Myasthenia Gravis (MG) is a chronic autoimmune disease characterized by muscle weakness and fatigue due to defective transmission at the neuromuscular junction. Typically, symptomatic assessments occur during clinical encounters, either as part of routine treatment assessments or in acute situations such as myasthenic crises. However, for a comprehensive understanding of mechanisms in MG and disease activity, continuous monitoring is essential.
Utilization of digital biomarkers derived from wearable devices offers unprecedented insights into diseases like MG and allows us to establish correlations between digital measures, clinical scores such as the QMG scale and MGC, as well as various questionnaires addressing sleep quality, quality of life or activities of daily living.
Data recorded by the used wearables (Withings Scanwatch 2) cover various parameters including activity-related data (step count, minutes in certain intensity levels), basic cardiovascular measurements such as heart rate, and sleep-related data (total time asleep, sleep quality, etc.).
Prospectively, this study aims at improving patient care by gaining a deeper insight into Myasthenia Gravis and its dynamic disease activity while concurrently monitoring the efficacy and safety of treatments, particularly of the newly approved drugs for MG.
In the future, wearables might hold the potential to improve treatment processes and optimize therapeutic approaches. For instance, digital biomarkers could serve as early warning signs for phenomena like heightened disease activity or end-of-dose phenomena, paving the way for personalized treatment strategies tailored to individual patient needs.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- 未提供
排除标准
- •Patients who meet any of the following criteria will be excluded from participation:
- •Unable or unwilling to give informed consent
- •Unable or unwilling to use the smartphone app
- •Any significant comorbidity that might potentially interfere with the ability to successfully participate in the study, based on investigator´s judgment
- •Patient with exclusively ocular symptoms (ocular myasthenia gravis)
结局指标
主要结局
MGFA-PIS: Myasthenia Gravis Post intervention status
时间窗: Baseline, after 3 and 6 months
The MGFA-PIS is a scoring system used to assess the status of patients with MG. It divides MG presentations into different classes, classifying outcome measures and treatment effectiveness. It has 8 classes; Minimal Manifestation can be further described within four dimensions.
MG-ADL: Myasthenia Gravis Activities of Daily Living
时间窗: Baseline, after 3 and 6 months
The MG-ADL is a tool used to assess the functional status of patients with MG in their daily activities. It consists of questions related to various activities of daily living such as speaking, chewing, swallowing, walking etc. It ranges from 0 to 24, with higher scores indicating a greater impairment in daily life.
MG-QoL15: Myasthenia Gravis Quality of Life-15
时间窗: Baseline, after 3 and 6 months
The MG-QoL assesses the quality of life in patients with MG, covering various aspects of daily life such as physical functioning, social interactions, emotional well-being and overall satisfaction with life. It ranges from 0 to 60, with higher scores indicating a greater impact of MG on life quality.
MGC: Myasthenia Gravis Composite
时间窗: Baseline, after 3 and 6 months
The MGC is a clinical tool to evaluate the severity and progression of MG. It ranges from 0 to 50, with higher scores indicating a higher severity of MG.
QMG: Quantitative Myasthenia Gravis Scale
时间窗: 6 months, monthly
The QMG-Score is a clinical tool to assess the severity of symptoms in MG. It evaluates muscle strength and function in specific muscle groups (ocular, oral, facial, neck and limb muscles), grip strength and vital capacity. It ranges from 0 to 39, with higher scores indicating an increased symptom load.
次要结局
- 10. Questionnaire on Smartwatch Usage (after 6 Months) containing the System Usability Scale(After 6 months)
- Digital Measurements: Activity parameters - longitudinal development of approximate distance travelled (meter)(6 months, continuously)
- Digital Measurements: Activity parameters - longitudinal development of approximate calories burned as defined by Withings(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of time awake (seconds)(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of time to sleep (seconds)(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of total time asleep (seconds)(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of time spent in bed before falling asleep (seconds)(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of Withings sleep Score as defined by Withings(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of heart rate variability (milliseconds)(6 months, continuously)
- HADSD: Hospital Anxiety and Depression Scale(Baseline, after 3 and 6 months)
- Digital Measurements: Activity parameters - longitudinal development of duration of itnense activity (seconds) as defined by Withings(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of ratio of sleep/time in bed(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of time awake after first falling asleep (seconds)(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of average heartrate (beats/min)(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of time in moderate heartrate zone (seconds)(6 months, continuously)
- WHOQoL-BREF: World Health Organization Quality of Life Questionnaire Brief Version(Baseline, after 3 and 6 months)
- EoD-Questionnaire: End of Dose-Questionnaire(Baseline, after 3 and 6 months)
- Sleep-Questionnaire:(Baseline, after 3 and 6 months)
- Digital Measurements: Activity parameters - longitudinal development of duration of soft activity (seconds) as defined by Withings(6 months, continuously)
- Digital Measurements: Activity parameters - longitudinal development of active time (seconds) as defined by Withings(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of number of times user woke up(6 months, continuously)
- Digital Measurements: Sleep parameters - longitudinal development of total time in bed (seconds)(6 months, continuously)
- Digital Measurements: Activity parameters - Longitudinal development of step count(6 months, continuously)
- Digital Measurements: Activity parameters - longitudinal development of duration of moderate activity (seconds) as defined by Withings(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of time in light heartrate zone (seconds)(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of time in intense heartrate zone (seconds)(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of time in maximal heartrate zone (seconds)(6 months, continuously)
- Digital Measurements: Cardiovascular parameters - longitudinal development of maximal heartrate (beats/min)(6 months, continuously)
- Smartwatch adherence(Continously 6 motnhs)
- Digital Measurements: Cardiovascular parameters - longitudinal development of minimum heartrate (beats/min)(6 months, continuously)
研究者
Dr. med. Marc Günter Pawlitzki
Senior Physician
Heinrich-Heine University, Duesseldorf