Clinical trial NCT06002945: Exploring Outcomes and Characteristics of Myasthenia Gravis 2
Official title: Exploring Outcomes and Characteristics of Myasthenia Gravis 2
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Myasthenia Gravis
Keywords: Neuromuscular
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Muscle weakness
Autoimmune Diseases
Immune System Diseases
Lead sponsor: Yale University
Sponsor class: OTHER
Collaborators: name: National Center for Advancing Translational Sciences (NCATS)
name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2023-08-21
Enrollment: 400
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects will be 18 years of age or older with a clinical diagnosis of a Myasthenia Gravis.

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.

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.
* 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.

Primary outcomes:
measure: The number of participants enrolled and serial blood reported at the conclusion of the enrollment period.
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California-Irvine
state: California
city: Los Angeles
country: United States
facility: Cedars Sinai Medical Center
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Washington
country: United States
facility: George Washington University
state: District of Columbia
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University
state: North Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
affiliation: Yale University
name: Richard Nowak, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Duke University
name: Vern Juel, MD
role: PRINCIPAL_INVESTIGATOR
References: