The Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis (ADAPT-teleMG)

• ClinicalTrials.gov Identifier: NCT05917184
• Recruitment Status: Completed
• First Posted: June 23, 2023
• Last Update Posted: June 23, 2023
• Sponsor: Yale University
• Collaborators: National Institute of Neurological Disorders and Stroke (NINDS); National Center for Advancing Translational Sciences (NCATS)
• Information provided by (Responsible Party): Richard Nowak, Yale University

Study Description

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.

Condition or disease	Intervention/treatment
Myasthenia Gravis	Other: Telehealth

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 52 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: The Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis (ADAPT-teleMG)
• Actual Study Start Date: August 30, 2021
• Actual Primary Completion Date: January 20, 2023
• Actual Study Completion Date: January 20, 2023

Groups and Cohorts

Group/Cohort	Intervention/treatment
Neurological Outpatients; Participants in this group will be recruited from the outpatient clinical population at participating MGNet sites. Treating physicians will make the initial determination of patients' potential eligibility to participate in the study.	Other: Telehealth; Two video telehealth visits will be conducted and recorded within 3 days of one another (+/- 1 day)

Outcome Measures

Primary Outcome Measures :

1. Interrater reliability of the virtual Myasthenia Gravis Composite (MGC-v) score. [ Time Frame: 3 days (+/- 1 day) ]
   Interrater reliability of the virtual Myasthenia Gravis Composite (MGC-v) score will be estimated between two independent rater assessments performed on Visit 1 (V1) and Visit 2 (V2). The MGC score is a validated, patient-reported 10-item assessment tool for evaluating the symptoms and signs of MG. Patient assessment includes self-report of impact (normal, mild, moderate, or severe) and is additionally weighted for clinical significance. Total score ranges from 0 to 50, with higher scores indicating a greater impact of MG on functional activities. A three point change is considered clinical meaningful. This brief assessment takes approximately 10 minutes to complete.
2. Intrarater reliability (test-retest reliability) of the MGC-v score. [ Time Frame: 3 days (+/- 1 day) ]
   Interrater reliability of the virtual Myasthenia Gravis Composite (MGC-v) score will be estimated between two independent rater assessments performed on Visit 1 (V1) and Visit 2 (V2). The MGC score is a validated, patient-reported 10-item assessment tool for evaluating the symptoms and signs of MG. Patient assessment includes self-report of impact (normal, mild, moderate, or severe) and is additionally weighted for clinical significance. Total score ranges from 0 to 50, with higher scores indicating a greater impact of MG on functional activities. A three point change is considered clinical meaningful. This brief assessment takes approximately 10 minutes to complete.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• 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

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

Contacts and Locations

Locations

United States, Connecticut

Yale University

New Haven, Connecticut, United States, 06519

United States, District of Columbia

George Washington University

Washington, District of Columbia, United States, 20037

United States, Illinois

University of Chicago

Chicago, Illinois, United States, 60637

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

United States, North Carolina

Duke University Medical Center

Durham, North Carolina, United States, 27710

Sponsors and Collaborators

Yale University

National Institute of Neurological Disorders and Stroke (NINDS)

National Center for Advancing Translational Sciences (NCATS)

Investigators

• Principal Investigator: Richard Nowak, MD, MS; Yale University
• Principal Investigator: Amanda Guidon, MD, MPH; Massachusetts General Hospital

More Information

• Responsible Party: Richard Nowak, Principal Investigator, Yale University
• ClinicalTrials.gov Identifier: NCT05917184
• Other Study ID Numbers: 2000030599; 1U54NS115054-01 ( U.S. NIH Grant/Contract )
• First Posted: June 23, 2023
• Last Update Posted: June 23, 2023
• Last Verified: June 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Richard Nowak, Yale University:

Neuromuscular; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Muscle weakness; Autoimmune Diseases; Immune System Diseases; Neuromuscular Junction Diseases; Outcome measures

Additional relevant MeSH terms:

Myasthenia Gravis; Muscle Weakness; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Pathologic Processes; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases