Safety and Efficacy of BAFF-R CART Cell Therapy for Refractory Neuroimmune Diseases Evaluating Dose Escalation and Preliminary Outcomes

What this Trial Is About

Researchers are evaluating the safety, tolerability, and preliminary effectiveness of autologous T cells engineered to express a chimeric antigen receptor targeting the B-cell activating factor receptor BAFFR in patients with refractory neuroimmune diseases. This phase IbIIa dose-escalation study aims to find the maximum tolerated dose MTD of BAFF-R CART cells and then further assess efficacy at that dose, enrolling 12 patients per disease for dose escalation and 15 patients per disease for efficacy evaluation. Participants will receive BAFF-R CART cell therapy in escalating dose levels 0.5, 1, and 2 million CART cells per kilogram. Prior to the infusion of CART cells, patients undergo lymphodepletion with cyclophosphamide and fludarabine given intravenously over three days. The CART cells are infused intravenously on day 0 following the lymphodepletion. Dose escalation proceeds cautiously with close monitoring to determine the dose-limiting toxicity and maximum tolerated dose. Throughout the study, participants will be closely monitored for adverse events, vital signs, physical exams, lab tests, ECGs, and echocardiograms for up to two years after infusion. Researchers will assess disease-related clinical scores, MRI lesion changes, immune cell levels, and antibody changes at various points up to one year post-infusion. Long-term follow-up includes evaluation of CART cell levels and pharmacodynamics for up to 15 years. The total participation duration varies, with intensive early monitoring and extended long-term safety and efficacy assessments.

Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases