Phase I Trial of SYS6020 CAR-T Cell Injection for Safety and Early Effectiveness in Adults With Refractory Generalized Myasthenia Gravis

What this Trial Is About

Researchers are conducting a phase I, multi-center, open-label trial to study the safety, tolerance, and preliminary effectiveness of SYS6020 injection in adults with refractory systemic myasthenia gravis. This study aims to find the recommended dose for future research and explore how the product behaves in the body and its immune response. The trial includes two stages dose-escalation and dose-expansion phases, following a detailed schedule of screening, treatment, and follow-up periods without lymphodepleting chemotherapy. Participants undergo leukapheresis to collect blood cells to manufacture the SYS6020 injection, which contains CAR T-cells targeting BCMA. In the dose-escalation phase, 3 to 5 dose levels of SYS6020 will be tested, with participants receiving three doses at varying cell levels per kilogram of body weight. The Safety Monitoring Committee will review safety data to decide on dose escalation or continuation. In the dose-expansion phase, participants receive the recommended dose determined from the first phase. Throughout the trial, participants will be closely monitored with assessments including MG-ADL, QMG, MGC, MG-QoL 15R scales, and MGFA classifications to evaluate efficacy. Safety will be tracked via vital signs, physical exams, lab tests, cytokines, ECGs, and recording of adverse events and concomitant treatments. Blood samples will be collected for pharmacokinetic and immunogenicity testing. The study includes a 28-day observation period after dosing, a six-month primary follow-up, and a long-term follow-up lasting up to five years, providing extensive safety and efficacy data over time.

A Study to Evaluate the Safety and Preliminary Efficacy of SYS6020 CAR T-cells in Patients With Refractory Generalized Myasthenia Gravis