View clinical trials related to Myasthenia Gravis, Generalized.
Filter by:Myasthenia gravis is an autoimmune neurological disease caused by autoantibodies primarily directed against components of the postsynaptic membrane of the neuromuscular junction. Approximately 85% of patients have antibodies directed against the acetylcholine receptor (anti-AChR). Anti-AChR antibodies act through three distinct mechanisms: 1. Activation of the classical complement pathway: Formation of membrane-attack complexes (MACs) results in the destruction of the postsynaptic membrane. 2. Mechanical blockade: Anti-AChR antibodies block the acetylcholine binding site on its receptor. 3. Internalization and lysosomal degradation: Bivalent IgG causes cross-linking of adjacent receptors leading to internalization and degradation of AChRs (antigenic modulation). Patient mortality has significantly reduced due to effective treatments preventing severe exacerbations of myasthenic symptoms. In the past five years, the FDA and EMA have approved complement inhibitors for the treatment of generalized myasthenia gravis with anti-AChR antibody positivity. Eculizumab, a humanized monoclonal antibody, binds to the complement fragment C5, inhibiting its cleavage into C5a and C5b, and preventing the formation of the terminal complement complex C5b-9 (MAC). Currently, Eculizumab is approved in Italy for generalized myasthenia gravis associated with anti-acetylcholine receptor antibody positivity. This class of drugs is generally more effective than conventional immunosuppressive therapies, though it comes with higher costs. There is heterogeneity among patients in their response to complement inhibitor therapies. Currently, there is no specific evidence indicating which patients may benefit most from this class of treatments. Personalized therapy, considering the predominant pathogenic mechanisms of anti-AChR in individual patients, seems necessary. Interindividual heterogeneity in the autoantibody repertoire could underlie different responses to complement inhibitor therapies. For example, inhibition of the complement cascade in patients whose autoantibodies also block receptors might result in an unsatisfactory treatment response. Moreover, C5 gene polymorphisms could explain a lack of response to these new drugs. Investigating the immune, genetic, and cellular profile of myasthenic patients eligible for these new pharmacological therapies could be useful for identifying predictive markers of response and personalizing therapeutic choices.
The purpose of this Phase 2 study is to evaluate the safety, tolerability, pharmacometrics, and efficacy of DNTH103 in participants with generalized myasthenia gravis (gMG).
The primary objective of this phase III trial is to investigate if Rituximab can reduce patients' functional impairment caused by MG. The secondary objectives of this trial are to assess whether treatment with rituximab in patients with MG will: - Allow faster and greater corticosteroid tapering - Reduce the frequency of exacerbations - Improve quality of life - Offer an acceptable safety and tolerability profile.
The purpose of this study is to evaluate the efficacy and safety of Telitacicept in the treatment of patients with generalized myasthenia gravis.
To evaluate the safety and efficacy of tocilizumab in the treatment of generalized myasthenia gravis (gMG) as an extension study for the participants who previously completed Study tMG(NCT05067348).
This study aims to characterize the clinical features, frequency of different subgroups of MG, and identify predictors of treatment responsiveness among different subgroups of MG. The predictors are including primary outcome (percentage of changes in MG scales at baseline at time of enrollment and after 3 months) and secondary outcome (treatment-related adverse events). Also it aims to determine the frequency of patients with refractory MG. This information will be used to understand the trends and mechanisms of disease relapse, and optimal management strategies.
Randomized, double-blind, placebo-controlled, parallel-group study with optional open-label extension.
This is a 6 month study that will evaluate the efficacy of 5mg tofacitinib tablet taken twice a day in patients with refractory myasthenia gravis. Twenty adult participants will receive a four month treatment of tofacitinib followed by elution for one month.
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis