Actively Recruiting
A Phase I Study to Evaluate BCMA-targeted Chimeric Antigen Receptor T Cell (SYS6020 Injection) in Patients With Refractory Generalized Myasthenia Gravis
Led by CSPC ZhongQi Pharmaceutical Technology Co., Ltd. · Updated on 2025-08-06
50
Participants Needed
1
Research Sites
230 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a phase I clinical trial to assess the safety and tolerance of SYS6020 injection in adults with refractory generalized myasthenia gravis. This study aims to determine the recommended dose for future research and preliminarily evaluate the treatment's clinical effects, as well as explore its behavior in the body and immune response. The trial is open-label and involves multiple centers, focusing on patients who have not responded to previous treatments. The trial consists of two phases: a dose-escalation phase exploring 3 to 5 dose levels and a dose-expansion phase using the recommended dose identified. Participants undergo leukapheresis to collect blood cells for the SYS6020 injection, which consists of autologous CAR-T cells targeting BCMA. Each participant receives three doses of SYS6020 injection, with dosing intervals of seven days. No lymphodepleting chemotherapy is used. Safety decisions are guided by a Safety Monitoring Committee throughout the dose-escalation. Participants are involved in several study periods, including screening, leukapheresis, pre-dosing assessments, treatment infusions, a 28-day safety observation, a six-month primary follow-up, and a long-term follow-up lasting up to five years. Researchers evaluate efficacy using several myasthenia gravis clinical scales and antibody levels, while safety is monitored through vital signs, physical exams, lab tests, ECGs, and adverse event tracking. Blood samples are collected to study pharmacokinetics and immunogenicity throughout the trial.
CONDITIONS
Brief Title
A Study to Evaluate the Safety and Preliminary Efficacy of SYS6020 CAR T-cells in Patients With Refractory Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 65 years old
- Diagnosed with generalized myasthenia gravis (MGFA clinical classification II-IV)
- Diagnosed with refractory myasthenia gravis
- QMG score greater than 11 during screening and before leukapheresis
- Positive acetylcholine receptor antibody (AChR-Ab) and/or muscle-specific receptor tyrosine kinase (MuSK) antibody
- Daily glucocorticoid dose stable and not exceeding 40mg prednisone or equivalent for at least 4 weeks prior to baseline
- Understands the trial and voluntarily signs informed consent
You will not qualify if you...
- Known allergy or intolerance to SYS6020, its components, or related drugs
- Major uncontrolled chronic diseases increasing risk
- Other autoimmune diseases requiring systemic treatment (except stable autoimmune thyroid disease with normal function)
- Severe recurrent or active infection affecting participation
- Positive HIV, hepatitis B surface antigen, or elevated HBV-DNA levels
- History of malignant tumors within 5 years or active tumors (some thymoma subtypes allowed)
- Serious respiratory or cardiovascular diseases
- Clinically significant abnormal lab results (e.g., elevated liver enzymes, kidney function, coagulation)
- Alcohol dependence or drug abuse
- Current psychotic disorder affecting adherence
- History of primary immunodeficiency or prior organ/bone marrow transplant, or planned transplant
- Recent significant bleeding or long-term anticoagulant use
- Previous CAR-T or gene therapy
- Recent intravenous immunoglobulin, plasmapheresis, plasma separation, or hemodialysis within 1 month before leukapheresis
- Recent use of certain immunosuppressants or targeted biological agents before dosing
- Any condition deemed unsuitable by the investigator for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 3 weeks
Participants receive 3 doses of SYS6020 injection, an autologous CAR-T cell therapy targeting BCMA, administered at 7-day intervals.
3 dosing visits (in-person) spaced 7 days apart
Duration - Up to 12 months
Participants are monitored for safety, efficacy, and adverse events up to 12 months after dosing.
Regular visits for monitoring and assessments during 12 months post-treatment
Trial Site Locations
Total: 1 location
1
Qilu Hospital, Shandong University
Jinan, China
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
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