Clinical trial NCT05917184: The Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis
Official title: The Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis (ADAPT-teleMG)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myasthenia Gravis
Keywords: Neuromuscular
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Muscle weakness
Autoimmune Diseases
Immune System Diseases
Neuromuscular Junction Diseases
Outcome measures
Lead sponsor: Yale University
Sponsor class: OTHER
Collaborators: name: National Institute of Neurological Disorders and Stroke (NINDS)
name: National Center for Advancing Translational Sciences (NCATS)
First posted: 2023-06-23
Enrollment: 52
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: Approximately 50 adult participants with a confirmed diagnosis of MG will be enrolled with a range of disease activity from approximately 7 MGNet clinical study sites

Brief summary: The goal of this pilot study is to determine the reliability of myasthenia gravis (MG) specific outcome measures obtained during virtual encounters with patients with myasthenia gravis. The main question it aims to answer is: Are MG-specific outcome measures obtained during virtual encounters reliable?

Participants will complete two virtual study visits in which they will be administered common MG-specific outcome measures and a newly developed outcome measure developed specifically for telemedicine assessments of MG patients.

Detailed description: The purpose of this study is to better understand the use of modified clinical assessments during telehealth visits for patients with Myasthenia Gravis (MG). The study will consist of 2 virtual telemedicine visits. A screening visit will be conducted to optimize the telemedicine environment, provide training on the telemedicine platform being used in the study, and collect demographic, medical history, and medication information. Participants who meet eligibility requirements will have a telemedicine visit (Visit 1) with a study doctor where assessments adapted for the telemedicine environment will be performed. Participants will also be asked to fill out questionnaires related to MG or their experience with telemedicine either online or with the study investigators. A second visit (Visit 2), conducted within 3 days (+/- 1 day), will replicate the assessments performed in V1. Visit 1 and Visit 2 will be recorded for later review by study team members. The investigators hope to establish the effectiveness of these clinical tools in order to better manage MG in the future in clinical care and research study visits.

Eligibility criteria: Inclusion Criteria:

* Has the capacity to understand and sign an informed consent form
* Diagnosed with acquired autoimmune MG based on clinical syndrome and seropositivity for MG associated autoantibodies (AChR binding, MuSK, or LRP4 antibodies), and/or a decremental response on repetitive nerve stimulation, positive single-fiber electromyography or positive edrophonium test
* Subject has been previously evaluated in a face-to-face clinical encounter at one of the participating study sites within the prior 3 years
* Primary language is English, or equivalent level of English language fluency
* Required disease severity range from asymptomatic-mild to moderate-severe as previously judged by the Investigator.

Exclusion Criteria:

* Inadequate technology for telehealth visits as assessed by the Investigator (e.g., no internet connection, no laptop/tablet with video capability, etc). Cellular phones are not acceptable. Subjects must use a laptop or tablet.
* Is unable or unwilling to comply with the study procedures, including video recording, or telehealth visit.
* Any significant medical/psychiatric condition that, in the opinion of the Investigator, may interfere with interpretation of subject evaluation(s) or protocol compliance
* Known pregnancy
* MGFA severity class IVb or V
* Concurrent participation in an interventional clinical trial

Primary outcomes:
measure: Interrater reliability of the virtual Myasthenia Gravis Composite (MGC-v) score.
timeFrame: 3 days (+/- 1 day)
measure: Intrarater reliability (test-retest reliability) of the MGC-v score.
timeFrame: 3 days (+/- 1 day)
Secondary outcomes:
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Washington
country: United States
facility: George Washington University
state: District of Columbia
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Yale University
name: Richard Nowak, MD, MS
role: PRINCIPAL_INVESTIGATOR
affiliation: Massachusetts General Hospital
name: Amanda Guidon, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: