Abstract Details

Title A prospective natural history study and biorepository for patients with myasthenia gravis (EXPLORE-MG2)

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P6 - Poster Session 6 (5:30 PM-6:30 PM)

Poster/Presentation
Number 11-005

Background The MG Rare Disease Clinical Research Network (MGNet) was funded by the National Institutes of Health (NIH) to gain better understanding of the clinical course of MG and develop improved approaches to diagnosis and treatment. As part of this initiative a multicenter prospective natural history study and biorepository was developed: Exploring Outcomes and Characteristics of Myasthenia Gravis 2 (EXPLORE-MG2).

Objective To define clinical features, disease course, and collect biospecimens in patients with myasthenia gravis (MG), including understudied subgroups such as seronegative, muscle-specific kinase antibody (MuSK), and LRP4 antibody positive MG.

Design/Methods EXPLORE-MG2 is a web-based observational registry that incorporates NIH recommended common data elements for MG. Key eligibility criteria include: ≥18 years old; diagnosed with MG within 2-years of study enrollment based on clinical presentation and seropositivity for MG associated autoantibodies, and/or abnormal neurophysiology test or positive edrophonium test. Biospecimen collection focuses on immunosuppressive naïve patients and rare MG subgroups. Participants will be followed for at least 2-years with study visits occurring approximately every 6 months in the context of usual clinical care. The study has been open to enrollment since January 2021 with 6 sites currently activated/participating.

Results A total of 62 patients have been enrolled and 152 biospecimens were collected as of 10/1/2021. The mean age was 57 years (range 20-84); 47% were female, 66% were acetylcholine receptor antibody-positive, 16% were MuSK MG, and 18% were seronegative. Enrollment of new participants and follow-up of existing participants are ongoing to reach our current goal of 400 enrolled participants. We will present updated enrollment data and demographics at the meeting.

Conclusions The EXPLORE-MG2 study is active after a COVID-19 pandemic related delay. Samples and clinical data will be available to researchers for current and future investigation. Data from EXPLORE-MG2 will improve clinical trial readiness for future studies and facilitate development of treatment-responsive biomarkers.

Authors/Disclosures
Jeff Guptill, MD, FAAN (argenx US) PRESENTER	Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has stock in argenx.
Richard J. Nowak, MD (Yale University School of Medicine)	Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Viela Bio. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cour Pharma. The institution of Dr. Nowak has received research support from Ra Pharma. The institution of Dr. Nowak has received research support from Alexion . The institution of Dr. Nowak has received research support from Momenta . The institution of Dr. Nowak has received research support from Immunovant . Dr. Nowak has received research support from argenx. Dr. Nowak has received research support from Viela Bio . Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities.
Amanda C. Guidon, MD (Massachusetts General Hospital)	An immediate family member of Dr. Guidon has received personal compensation for serving as an employee of GE Healthcare. Dr. Guidon has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Guidon has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Momenta. Dr. Guidon has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Guidon has received research support from MGFA. The institution of Dr. Guidon has received research support from Project Data Sphere. The institution of Dr. Guidon has received research support from MGNet. The institution of Dr. Guidon has received research support from MGNet. Dr. Guidon has received publishing royalties from a publication relating to health care.
James F. Howard, Jr., MD, FAAN (The University of North Carolina, Dept of Neurology, CB 7025)	Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for arenx . Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ra Pharma (now UCB Biosciences). Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi US. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Academic CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for PeerView CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Platform Q CME. Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Zai Laboratories. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Medscape CME. Dr. Howard has stock in General Electric dividends. Dr. Howard has stock in Johnson & Johnson dividends. Dr. Howard has stock in Pfizer dividends. An immediate family member of Dr. Howard has stock in GlaxoSmithKline dividends. An immediate family member of Dr. Howard has stock in GE Healthcare dividends. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx . The institution of Dr. Howard has received research support from UCB Biosciences. The institution of Dr. Howard has received research support from NIH. The institution of Dr. Howard has received research support from Centers for Disease Control/Research Triangle Institute. The institution of Dr. Howard has received research support from Duke University (DCRI). The institution of Dr. Howard has received research support from Cartestian Therapeutics. Dr. Howard has a non-compensated relationship as a Scientific Advisiory Board member, Committee member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities.
Betty C. Soliven, MD (University of Chicago)	Dr. Soliven has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CVS Pharmacy. The institution of Dr. Soliven has received research support from NIH. The institution of Dr. Soliven has received research support from NIH. The institution of Dr. Soliven has received research support from Alexion. The institution of Dr. Soliven has received research support from Roche/Genentech.
	No disclosure on file
	No disclosure on file
Yingkai Li, MD, PhD (Duke University)	Dr. Li has nothing to disclose.
	No disclosure on file
Immaculada Aban	No disclosure on file
Gary R. Cutter, PhD (University of Alabama At Birmingham)	Dr. Cutter has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biodelivery Sciences International, Biogen, Click Therapeutics, Genzyme, Genentech, GW Pharmaceuticals, Immunic, Klein-Buendel Incorporated, Medimmune/Viela Bio, Medday, Merck/Serono, Neurogenesis LTD, Novartis, Osmotica Pharmaceuticals, Perception Neurosciences, Recursion/Cerexis Pharmaceuticals, Regeneron, Reckover Pharmaceuticals, Roche, TG Therapeutics.. Dr. Cutter has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics, AI therapeutics, AMO Pharma, Astra-Zeneca, Avexis Pharmaceuticals, Biolinerx, Brainstorm Cell Therapeutics, Bristol Meyers Squibb/Celgene, CSL Behring, Galmed Pharmaceuticals, Green Valley Pharma, Horizon Pharmaceuticals, Immunic, Karuna Therapeutics, Mapi Pharmaceuticals LTD, Merck, Mitsubishi Tanabe Pharma Holdings, Opko Biologics,Prothena Biosciences, Novartis, Regeneron, Sanofi-Aventis, Reata Pharmaceuticals, NHLBI (Protocol Review Committee), University of Texas Southwestern, University of Pennsylvania, Visioneering Technologies, Inc.. Dr. Cutter has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for JASN.
Henry J. Kaminski, MD, FAAN (George Washington University)	Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Kaminski has received personal compensation in the range of $0-$499 for serving as a Consultant for Cabaletta Bio. The institution of Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Kaminski has stock in mimivax. The institution of Dr. Kaminski has received research support from National Institutes of Health. Dr. Kaminski has received publishing royalties from a publication relating to health care.
Jeff Guptill, MD, FAAN (argenx US)	Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has stock in argenx.