Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia...
Myasthenia GravisThis is a multicenter, randomized, double blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of tolebrutinib compared with placebo in adult participants aged 18 to 85 years old with moderate-to-severe gMG, who are receiving Standard of Care (SoC). The double-blind (DB) treatment period of 26 weeks will comprise of 7 site visits followed by a 2-year open label extension (OLE) period with quarterly visits. The efficacy of tolebrutinib versus placebo during DB period will be assessed by clinical evaluations, that include scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs). These evaluations will continue during the OLE in order to measure long term efficacy and safety.
Trial of Orencia in Patients With Myasthenia Gravis
Myasthenia GravisThis pilot research study is being done to see if the drug abatacept (Orencia ®) will be helpful in treating patients with myasthenia gravis (MG) who do not respond satisfactorily to other drugs that are used to suppress the immune system. Abatacept has been successful in treating experimental MG in laboratory animals, and this study is to determine its effectiveness in patients with MG.
Serum Auto-Antibodies in Neurological Diseases
Multiple SclerosisMyasthenia Gravis1 moreUnder normal conditions our immune system protects us against infections and tumors. The immune system does this by recognizing that the infecting organism or the tumor is foreign to the body and attacking it. One way the immune system attacks a foreign target is by making proteins called antibodies that bind to the target. Sometimes, for reasons we poorly understand, the immune system wrongly identifies part of our own body as being foreign and attacks it. This can result in disease such as some forms of diabetes and thyroid disease, as well as some neurological diseases. In this study, one tablespoon of blood will be removed from each subject and tested to see if the immune system is making antibodies against components of the nerves and muscles. We also hope to learn if these antibodies contribute to the development or worsening of illnesses of the nervous system. Only one blood draw is required, but subjects may be asked to give up to 8 additional blood samples to see if the level of antibodies changes over time. Any additional blood draws would be performed at regularly scheduled clinic visits. There would be at least 3 months between blood draws over a period of up to 3 years, if requested by the physician. Depending on your diagnosis, the physician may also request the collection of mouth (buccal) cells. This takes about one minute and is painless. The cells are collected by swishing a swab around your mouth. This cheek swab would be done with each blood draw. Please note that this study is conducted ONLY at UC Davis and that all participants must be seen in our clinic located in Sacramento, CA. Results of the testing performed in this study are not given to the participants. This study is not intended to treat or diagnose any condition.
Therapy of Antibody-mediated Autoimmune Diseases by Bortezomib (TAVAB)
Myasthenia GravisSystemic Lupus Erythematosus1 moreThe aim of this pilot study is to investigate the application of proteasome inhibitor Bortezomib (Velcade®, approved for therapy of multiple myeloma) in patients with therapy-refractory antibody-mediated autoimmune diseases. The investigators hypothesis is that the proteasome inhibition will lead to reduced antibody titers and improved clinical outcome.
Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis
Myasthenia GravisMG may be neonatal, congenital, or autoimmune. Neonatal MG arises from transplacental transfer of ACh receptor antibodies from a mother with autoimmune MG to the fetus. Neonatal MG resolves with post delivery clearance of maternal antibodies. Congenital MG results from a genetic defect in the ACh receptor. Patients with congenital MG do not have ACh receptor antibodies. Both neonatal and congenital MG are excluded from this study. Autoimmune MG, which is the most common form of MG, affects approximately 25,000 Americans. Like most autoimmune diseases, it is associated with particular HLA genotypes, has a female predominance, and environmental factors involved in breaking tolerance to the ACh receptor are unknown. Patients with refractory and severe autoimmune MG will be considered candidates for this study. The purpose of this study is to assess the toxicity/feasibility (phase I) of autologous hematopoietic stem cell transplantation for refractory myasthenia gravis.
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult...
Generalized Myasthenia GravisThe purpose of the MycarinGstudy is to evaluate the long-term safety, tolerability and long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG).
A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia...
Generalized Myasthenia GravisThe purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).
A Study to Evaluate the Efficacy, Safety and PD and PK of HBM9161 in MG Patients
Myasthenia GravisPrimary Objectives: To investigate the efficacy of HBM9161 in patients with attack of MG in China
BeatMG: Phase II Trial of Rituximab In Myasthenia Gravis
Myasthenia GravisThe specific primary objective of this study is to determine whether rituximab is a safe and beneficial therapeutic for Myasthenia Gravis (MG) that warrants further study in a phase III efficacy trial.
Efficacy and Safety Study of GB-0998 for Treatment of Generalized Myasthenia Gravis
Generalized Myasthenia GravisThis randomized controlled, multi-center study will carry out to assess the efficacy of GB-0998 compared to plasmapheresis in the treatment of the generalized Myasthenia Gravis based on the changes in Quantitative Myasthenia Gravis score (QMG score) as primary endpoint, and in addition, to assess the safety of GB-0998