An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod...
Generalized Myasthenia GravisThe purpose of this open-label study is to investigate the efficacy, safety, and tolerability of a continuous regimen of efgartigimod compared with a cyclic regimen in participants with Generalized Myasthenia Gravis (gMG). Study details include: The study duration will be up to 138 weeks (including screening and a safety follow-up of up to 9 weeks) Part A (regimen comparison period) - 21 weeks Part B (extension period) - up to 105 weeks The visit frequency, including virtual visits, will be weekly through Week 21 and every 5 weeks for the remainder of the study.
Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously...
Generalized Myasthenia GravisThe purpose of this study is to evaluate the long-term safety and tolerability of efgartigimod PH20 SC 1000 mg, and the clinical efficacy, PD, pharmacokinetics (PK), immunogenicity, impact on the quality of life (QoL) of the participants, treatment satisfaction, and administration method preference, and the feasibility of self- and caregiver-supported administration of the SC injection. Treatment duration: 3-week treatment periods, repeated as needed with at least 28 days in between treatment periods Health measurements: total levels of immunoglobulin G (IgG), Acetylcholine receptor binding autoantibodies (AChR-Ab) levels, Myasthenia Gravis Activities of Daly Living (MG-ADL).
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia...
Myasthenia GravisMuSKEvaluate the long-term safety of amifampridine phosphate in the symptomatic relief of antibody positive MuSK-MG.
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia...
Generalized Myasthenia GravisThe purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis
Myasthenia GravisThis study is designed to evaluate the effectiveness and safety of tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy.
Descriptive Analysis of Real-world Data Collected With ME&MGopen
Myasthenia GravisThe ME&MGopen smartphone application is an investigational software for research purposes only, developed by Ad Scientiam. It features digital tests to assess the respiratory capacity ("My Breathing" Test), dysarthria ("My Voice" Test), ptosis ("My eyelids" test), as well as upper and lower limb muscle function ("My arms" Test and "My legs" Test). The mobile app also includes e-questionnaires related to activities of daily living, pain, insomnia, quality of life and depression. The objectives of the study are to collect data on patients' symptoms with the application in a real life setting, to assess adherence to the use of the tool, user experience and satisfaction with the application, and safety of use.
A Prospective Cohort Study of Myasthenia Gravis in China
Myasthenia GravisThe goal of this prospective cohort study is to investigate long-term therapeutic strategies for myasthenia gravis (MG) and identify potential biomarkers. The main questions it aims to answer are: Whether low-dose oral steroids may lead to a reduction in the recurrence rate among patients with MG. To identify potential biomarkers that can predict disease progression and prognosis. This study recruits well-controlled patients with MG. Based on patient preferences and considerations such as coexisting conditions (e.g., uncontrolled hypertension, diabetes, severe osteoporosis, obesity), the participants will be non-randomly divided into two groups: a maintenance steroid therapy group and a withdrawal group (withdraw all immunosuppresants). Subsequently, these groups of patients will undergo long-term follow-up assessments.
Clinical Prognosis and Progression of Myasthenia Gravis Patients
Myasthenia GravisThis study collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG.
Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies...
Generalized Myasthenia GravisLong-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).
TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world...
Ocular Myasthenia GravisThis study collects the clinical data of new-onset ocular myasthenia gravis (OMG) patients, assesses outcomes and adverse effects of different treatment options, and evaluate risk factors of conversion to generalized MG(GMG).