Exploring Outcomes and Characteristics of Myasthenia Gravis 2

Active, not recruiting

• Conditions: Myasthenia Gravis
• Interventions: Other: Blood sample

• Registration Number: NCT06002945
• Lead Sponsor: Yale University

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.

Study Timeline

• Study Start: 2021-01-12
• Study First Submitted: 2023-08-14
• Study First Submitted QC: 2023-08-14
• Study First Posted: 2023-08-21
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-04
• Last Update Posted: 2024-12-10
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 400

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.

• Known active malignancy at enrollment other than skin cancer, low grade prostate cancer, or cervical cancer in situ. Thymoma is not included in this exclusion criteria.

• 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Neurological Outpatients	Blood sample	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.

Primary Outcome Measures

Name	Time	Method
The number of participants enrolled and serial blood reported at the conclusion of the enrollment period.	5 years

Trial Locations

Locations (10)

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, 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