Clinical trial NCT06441825: Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis
Official title: Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myasthenia Gravis
Myasthenic Syndrome
Lambert-Eaton Myasthenic Syndrome
Lead sponsor: Heinrich-Heine University, Duesseldorf
Sponsor class: OTHER
First posted: 2024-06-04
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with myasthenic syndromes treated at the University Clinic Duesseldorf.

Brief 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.

Detailed 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.

Eligibility criteria: * 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)

Primary outcomes:
measure: QMG: Quantitative Myasthenia Gravis Scale
timeFrame: 6 months, monthly
measure: MGC: Myasthenia Gravis Composite
timeFrame: Baseline, after 3 and 6 months
measure: MGFA-PIS: Myasthenia Gravis Post intervention status
timeFrame: Baseline, after 3 and 6 months
measure: MG-ADL: Myasthenia Gravis Activities of Daily Living
timeFrame: Baseline, after 3 and 6 months
measure: MG-QoL15: Myasthenia Gravis Quality of Life-15
timeFrame: Baseline, after 3 and 6 months
Secondary outcomes:
measure: WHOQoL-BREF: World Health Organization Quality of Life Questionnaire Brief Version
timeFrame: Baseline, after 3 and 6 months
measure: HADSD: Hospital Anxiety and Depression Scale
timeFrame: Baseline, after 3 and 6 months
measure: EoD-Questionnaire: End of Dose-Questionnaire
timeFrame: Baseline, after 3 and 6 months
measure: Sleep-Questionnaire:
timeFrame: Baseline, after 3 and 6 months
measure: 10. Questionnaire on Smartwatch Usage (after 6 Months) containing the System Usability Scale
timeFrame: After 6 months
measure: Digital Measurements: Activity parameters - Longitudinal development of step count
timeFrame: 6 months, continuously
measure: Digital Measurements: Activity parameters - longitudinal development of approximate distance travelled (meter)
timeFrame: 6 months, continuously
measure: Digital Measurements: Activity parameters - longitudinal development of duration of soft activity (seconds) as defined by Withings
timeFrame: 6 months, continuously
measure: Digital Measurements: Activity parameters - longitudinal development of duration of moderate activity (seconds) as defined by Withings
timeFrame: 6 months, continuously
measure: Digital Measurements: Activity parameters - longitudinal development of duration of itnense activity (seconds) as defined by Withings
timeFrame: 6 months, continuously
measure: Digital Measurements: Activity parameters - longitudinal development of active time (seconds) as defined by Withings
timeFrame: 6 months, continuously
measure: Digital Measurements: Activity parameters - longitudinal development of approximate calories burned as defined by Withings
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of time awake (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of number of times user woke up
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of time to sleep (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of total time asleep (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of total time in bed (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of ratio of sleep/time in bed
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of time spent in bed before falling asleep (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of time awake after first falling asleep (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Sleep parameters - longitudinal development of Withings sleep Score as defined by Withings
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of average heartrate (beats/min)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of maximal heartrate (beats/min)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of minimum heartrate (beats/min)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of time in light heartrate zone (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of time in moderate heartrate zone (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of time in intense heartrate zone (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of time in maximal heartrate zone (seconds)
timeFrame: 6 months, continuously
measure: Digital Measurements: Cardiovascular parameters - longitudinal development of heart rate variability (milliseconds)
timeFrame: 6 months, continuously
measure: Smartwatch adherence
timeFrame: Continously 6 motnhs
Other outcomes:
Locations:
city: Duesseldorf
country: Germany
facility: Heinrich-Heine University, Duesseldorf
Study officials:
References: