Exploring Outcomes and Characteristics of Myasthenia Gravis 2

NCT ID: NCT06002945

Last Updated: 2024-12-10

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 400 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-01-12
• Study Completion Date: 2026-12-31

Brief Summary

The goal of this prospective observational study is to create a network repository of clinical data and biological samples to help researchers learn more about myasthenia gravis.

Detailed Description

The EXPLORE-MG2 (EXPLoring Outcomes and chaRactEristics of Myasthenia Gravis 2) study is a multi-center natural history study of myasthenia gravis (MG), a rare autoimmune disease characterized by weakness of the voluntary muscles. MG has a prevalence of approximately 14-40 per 100,000 people in the United States. There is no cure for MG, however, understanding the disease is crucial to pave the way for development of new therapies and a deeper mechanistic understanding to improve patient outcomes. Thus, EXPLORE-MG2 aims to accomplish comprehensive clinical phenotyping linked to bio-specimen collection in an effort to better understand disease characteristics and identify treatment predictive and responsive biomarkers. The primary purpose of this project is to create a network repository of data and biological samples to help researchers learn more about myasthenia gravis.

Conditions

Myasthenia Gravis

Keywords

Neuromuscular; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Muscle weakness; Autoimmune Diseases; Immune System Diseases

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Neurological Outpatients

Participants in this group will be recruited from the outpatient clinical population at MGNet participating sites. Treating physicians will make the initial determination of patients' potential eligibility to participate in the study.

Blood sample

Intervention Type: OTHER

Each blood draw will involve removing approximately 75 mL (5 tablespoons) of blood

Interventions

Blood sample

Each blood draw will involve removing approximately 75 mL (5 tablespoons) of blood

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Diagnosed with MG based on clinical presentation 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 (EMG) or positive edrophonium test. Signs and symptoms should not be better explained by another disease process.
• A) Diagnosed with MG within 2 years of study enrollment and with adequate records to document MG history (e.g., onset, MG meds) prior to enrollment at the discretion of the investigator.

or B) MuSK antibody, LRP4 antibody, or thymoma-associated MG at any point in the disease course. Patients with a diagnosis of MG more than 2 years prior to the first study visit who are expected to undergo a thymectomy may also be enrolled.

• Participant has the capacity to understand and sign a written informed consent form.
• Participant must be willing to complete the study and return for follow-up visits.

Exclusion Criteria

• A history of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue that would confound outcome measure assessments or any disorder that interferes with participation in this protocol.
• Participant is unable or unwilling to comply with the study procedures.
• Treatment with any investigational agent within 4 weeks of the baseline visit or 5 half-lives of the investigational drug (whichever is longer).

1. Participation in other observational studies is not exclusionary

2. It is anticipated that some participants enrolled in EXPLORE-MG2 may be enrolled in future interventional trials while active in EXPLORE-MG2. This is acceptable. The exclusion only applies to starting EXPLORE-MG2 when participation in an interventional trial is imminent or active.

3. If an EXPLORE-MG2 participant is later enrolled in an interventional trial, it will be at the investigator's discretion whether to continue collecting biosamples while the patient is participating in the interventional trial. Collection of natural history data should continue.

• Any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates or that may affect the interpretation of the results or render the participant not an appropriate study participant.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Center for Advancing Translational Sciences (NCATS)

NIH

Sponsor Role: collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

Yale University

OTHER

Sponsor Role: lead

Responsible Party

Richard Nowak

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Richard Nowak, MD

Role: PRINCIPAL_INVESTIGATOR

Yale University

Vern Juel, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

University of California-Irvine

Irvine, California, United States

Cedars Sinai Medical Center

Los Angeles, California, United States

Yale University

New Haven, Connecticut, United States

George Washington University

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

University of Chicago

Chicago, Illinois, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

University of North Carolina

Chapel Hill, North Carolina, United States

Duke University Medical Center

Durham, North Carolina, United States

Wake Forest University

Winston-Salem, North Carolina, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Countries

United States

Other Identifiers

1U54NS115054-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

2000026920

Identifier Type: -

Identifier Source: org_study_id