Myasthenia Gravis clinical trials at UC Irvine

The primary objective is: To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG) The secondary objectives of the study are: - To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran monotherapy on: - Clinician-assessed signs of myasthenia gravis (MG) and muscle strength - Daily functioning that is impacted by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy only). - Proportion of patients with improvements in daily function that is impacted by signs and symptoms of MG - Proportion of patients that have improvements in clinician-assessed signs of MG and muscle strength - Health related quality of life - Proportion of patients with minimal MG symptoms - Patient- and clinician-reported signs and symptoms of MG - To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran monotherapy - To assess the concentration of total pozelimab in serum - To assess the concentrations of cemdisiran and its metabolites in plasma - To assess the immunogenicity of pozelimab - To assess the concentration of total C5 in plasma - To assess the immunogenicity of cemdisiran - To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement activation

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

Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction. This phase 1 study is being conducted to evaluate the safety of various dosing regimens of an investigational cell therapy, MuSK-CAART, that can be given to patients with anti-MuSK antibody positive Myasthenia Gravis (MuSK MG), who have active disease. Various dosing regimens of MuSK-CAART alone, in combination with cyclophosphamide (CY), and in combination with CY and fludarabine (FLU) will be evaluated. Treatment with MuSK-CAART may potentially lead to complete and durable remission of disease.