Exploring Outcomes and Characteristics of Myasthenia Gravis 2

Myasthenia Gravis Clinical Trial

— EXPLORE-MG2  

Official title:

Exploring Outcomes and Characteristics of Myasthenia Gravis 2

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06002945
• Other study ID #: 2000026920
• Secondary ID: 1U54NS115054-01
• Status: Recruiting
• Start date: January 12, 2021
• Est. completion date: December 2026

Study information

• Verified date: August 2023
• Source: Yale University
• Contact: Alex Hammett
• Phone: 919-668-8432
• Email: alex.hammett[@]duke.edu
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: December 2026
• Est. primary completion date: December 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis

Intervention

Other:
• Blood sample
Each blood draw will involve removing approximately 75 mL (5 tablespoons) of blood

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of North Carolina	Chapel Hill	North Carolina
United States	Medical University of South Carolina	Charleston	South Carolina
United States	University of Chicago	Chicago	Illinois
United States	Duke University Medical Center	Durham	North Carolina
United States	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	Wake Forest University	Winston-Salem	North Carolina

Sponsors (3)

Lead Sponsor	Collaborator
Yale University	National Center for Advancing Translational Sciences (NCATS), National Institute of Neurological Disorders and Stroke (NINDS)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The number of participants enrolled and serial blood reported at the conclusion of the enrollment period.		5 years