Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis

Myasthenia Gravis Clinical Trial

— POWER-MG  

Official title:

Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06441825
• Other study ID #: MPGv1.0
• Status: Recruiting
• Start date: May 2, 2024
• Est. completion date: October 31, 2025

Study information

• Verified date: May 2024
• Source: Heinrich-Heine University, Duesseldorf
• Contact: Marc Pawlitzki, PD Dr. med.
• Phone: +49211 8117887
• Email: neuro-trials[@]med.uni-duesseldorf.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: October 31, 2025
• Est. primary completion date: October 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

• Formal diagnosis of generalized Myasthenia Gravis, with at least one of the following

criteria:

• Response to oral or intravenous administration of an acetylcholinesterase inhibitor
• Evidence of pathological decrement/ elevated jitter
• Evidence of Myasthenia Gravis-typical antibody (AChR, LRP4, MuSK, Titin) OR other diagnosed Myasthenic Syndrome
• Age = 18
• Usage of Smartphone with Android 8.1 (or higher) or IOS12.3 (or higher)
• Able to provide informed consent, based on investigator´s judgment Exclusion Criteria:

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)

Related Conditions & MeSH terms

• Lambert-Eaton Myasthenic Syndrome
• Muscle Weakness
• Myasthenia Gravis
• Syndrome

Intervention

Other:
• Increased multimodal observation including digital monitoring
Digital monitoring combined with repeated clinical assessments and retrospective analysis of serological markers

Locations

Country	Name	City	State
Germany	Heinrich-Heine University, Duesseldorf	Duesseldorf

Sponsors (1)

Lead Sponsor	Collaborator
Heinrich-Heine University, Duesseldorf

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	QMG: Quantitative Myasthenia Gravis Scale	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.	6 months, monthly
Primary	MGC: Myasthenia Gravis Composite	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.	Baseline, after 3 and 6 months
Primary	MGFA-PIS: Myasthenia Gravis Post intervention status	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.	Baseline, after 3 and 6 months
Primary	MG-ADL: Myasthenia Gravis Activities of Daily Living	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.	Baseline, after 3 and 6 months
Primary	MG-QoL15: Myasthenia Gravis Quality of Life-15	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.	Baseline, after 3 and 6 months
Secondary	WHOQoL-BREF: World Health Organization Quality of Life Questionnaire Brief Version	The WHOQOL-BREF questionnaire measures quality of life across 4 domains: Physical health, psychological health, social relationships and environment. It also includes one question on overall QOL and one on general health. The WHOQOL-BREF scores correlate highly (.89 or above) with WHOQOL-100 scores, and demonstrate good discriminant validity, content validity, internal consistency and test-retest reliability. The four WHOQOL-BREF domain scores will be used as main outcome measure. The measure is calculated by summing the point values for the questions corresponding to each domain and then transforming the scores to a 0-100 point interval, with higher scores corresponding to greater QOL.	Baseline, after 3 and 6 months
Secondary	HADSD: Hospital Anxiety and Depression Scale	The HADSD is a self-assessment questionnaire measuring levels of anxiety and depression in patients. It contains two subscales: one for anxiety and one for depression. Total scores range from 0 to 42, with higher scores indicatiBAng higher levels of anxiety or depression.	Baseline, after 3 and 6 months
Secondary	EoD-Questionnaire: End of Dose-Questionnaire	The EoD-Questionnaire assesses the effectiveness of a treatment at the end of its dosing interval and asks about an increase in symptoms.	Baseline, after 3 and 6 months
Secondary	Sleep-Questionnaire:	The Sleep-Questionnaire contain the Pittsburgh Sleep Quality Index (PSQI) as well as two Items on the perceived impact of the smartwatch on the patient´s sleep. The PSQI assesses the quality of sleep over a one-month interval and measures seven component scores: sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleep medication, and daytime dysfunction. Total score ranges from 0 to 21, with higher scores indicating a poorer sleep quality.	Baseline, after 3 and 6 months
Secondary	10. Questionnaire on Smartwatch Usage (after 6 Months) containing the System Usability Scale		After 6 months
Secondary	Digital Measurements: Activity parameters - Longitudinal development of step count		6 months, continuously
Secondary	Digital Measurements: Activity parameters - longitudinal development of approximate distance travelled (meter)		6 months, continuously
Secondary	Digital Measurements: Activity parameters - longitudinal development of duration of soft activity (seconds) as defined by Withings		6 months, continuously
Secondary	Digital Measurements: Activity parameters - longitudinal development of duration of moderate activity (seconds) as defined by Withings		6 months, continuously
Secondary	Digital Measurements: Activity parameters - longitudinal development of duration of itnense activity (seconds) as defined by Withings		6 months, continuously
Secondary	Digital Measurements: Activity parameters - longitudinal development of active time (seconds) as defined by Withings		6 months, continuously
Secondary	Digital Measurements: Activity parameters - longitudinal development of approximate calories burned as defined by Withings		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of time awake (seconds)		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of number of times user woke up		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of time to sleep (seconds)		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of total time asleep (seconds)		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of total time in bed (seconds)		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of ratio of sleep/time in bed		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of time spent in bed before falling asleep (seconds)		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of time awake after first falling asleep (seconds)		6 months, continuously
Secondary	Digital Measurements: Sleep parameters - longitudinal development of Withings sleep Score as defined by Withings		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of average heartrate (beats/min)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of maximal heartrate (beats/min)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of minimum heartrate (beats/min)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of time in light heartrate zone (seconds)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of time in moderate heartrate zone (seconds)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of time in intense heartrate zone (seconds)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of time in maximal heartrate zone (seconds)		6 months, continuously
Secondary	Digital Measurements: Cardiovascular parameters - longitudinal development of heart rate variability (milliseconds)		6 months, continuously
Secondary	Smartwatch adherence	Wearing time of smartwatch (daily)	Continously 6 motnhs