Myasthenia Gravis Clinical Trials

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. Read Less

FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic/digitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification. Read Less

The purpose of this study is to create a Yale University Department of Neurology Myasthenia Gravis (MG) registry that will be used for current and future research projects involving the study of Myasthenia Gravis. Read Less

The goal of this observational study is to learn about the experiences of people living with Myasthenia Gravis (MG) in the United States. The main questions it aims to answer are: * How and when are people with MG diagnosed? * What are the most common symptoms associated with MG? * What treatments are being used to treat MG? * What are the impacts of MG on activities of daily living, employment and quality of life? * What are the experiences with exacerbation, hospitalization and healthcare access for people with MG? Participants will answer a survey to enroll in the study, and be invited to fill out an update survey twice a year. Read Less

This is a phase II, single center, randomized, double-blind, placebo-controlled, study in patients with a diagnosis of anti-AchR antibody positive myasthenia gravis. Read Less

To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions. Read Less

ME\&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG. ME\&MG contains digital active tests for the assessment of ptosis, breathing, dysarthria, upper- and lower-limb (arms and legs) weakness, treatment follow-up, and validated e-questionnaires related to daily activities, pain, fatigue, sleep, and depression disorders. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone with ME\&MG versus the standard in-clinic testing, as well as to evaluate the safety of the solution, its usability and satisfaction. Read Less

The goal of this observational study is to evaluate the feasibility of using wearable sensor and digital technologies to measure motor and speech function in adults with autoimmune Myasthenia Gravis (MG). The main question\[s\] it aims to answer are: * To measure the correlation of sensor-based measures of motor function with existing outcome measures including the MG-ADL, MGQOL15r, QMG, MGComposite, and Neuro-QOL Fatigue scales. * To develop and validate tablet-based digital assessments of speech and facial expression and to compare with existing outcome measures. Participants will wear a pendant sensor for 7 days and then participate in tablet-based and in-person myasthenia-specific physical examinations. This will be performed in concert with routine care in the Massachusetts General Hospital MG clinic. Read Less

The MuSK myasthenia gravis 1000 study seeks to collect saliva samples from 1000 subjects with laboratory confirmed diagnosis of MuSK myasthenia to identify genetic variations associated with MuSK MG. The data collected may be used by researchers to gain a better understanding of the cause of MuSK MG and to identify biomarkers and targeted therapy for MuSK MG. Read Less