The Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis

NCT05917184 | Completed

• 2 other identifiers: 20000305991U54NS115054-01
• Study Type: observational
• Target: 52
• Locations: 1 country
• Sites: 5

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.

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

Outcome Measures

Primary Outcomes (2)

• Interrater reliability of the virtual Myasthenia Gravis Composite (MGC-v) score.

  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.

  3 days (+/- 1 day)

• Intrarater reliability (test-retest reliability) of the MGC-v score.

  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.

  3 days (+/- 1 day)

Study Arms (1)

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

Interventions

Telehealth OTHER

Two video telehealth visits will be conducted and recorded within 3 days of one another (+/- 1 day)

Neurological Outpatients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Yale University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator
• National Center for Advancing Translational Sciences (NCATS): collaborator

Study Sites (5)

Yale University

New Haven, Connecticut, 06519, United States

Location

George Washington University

Washington D.C., District of Columbia, 20037, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Myasthenia Gravis; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Muscle Weakness; Autoimmune Diseases; Immune System Diseases; Neuromuscular Junction Diseases

Interventions

Telemedicine

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Delivery of Health Care; Patient Care Management; Health Services Administration

Study Officials

• Richard Nowak, MD, MS

  Yale University

  PRINCIPAL INVESTIGATOR

• Amanda Guidon, MD, MPH

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 14, 2023
• First Posted: June 23, 2023
• Study Start: August 30, 2021
• Primary Completion: January 20, 2023
• Study Completion: January 20, 2023
• Last Updated: June 23, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)