Actively Recruiting
A Study to Evaluate the Safety and Preliminary Efficacy of SYS6020 CAR T-cells 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
433 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study is a single-arm, open, 2-stage (dose-escalation phase and dose-expansion phase), multi-center, phase I clinical trial to evaluate the safety and tolerance of SYS6020 injection in the participants with refractory systemic myasthenia gravis, and determine the recommended dose (RD) for subsequent studies of the product, and to preliminarily evaluate the clinical efficacy of the product, as well as to explore the pharmacokinetics and immunogenicity of the product in vivo. The dose-escalation phase and dose-expansion phase include 7 periods, and they are respectively in sequence as follows: the screening period, apheresis period, pre-dosing assessment, SYS6020 injection infusion, DLT observation period, the primary follow-up period (6 months), and the long-term follow-up period (5 years). The DLT observation period is 28 days after receiving SYS6020 injection. The participants will not undergo lymphodepleting chemotherapy. The efficacy and safety profile of the participants will be continuously assessed during the trial. Efficacy measurement includes the MG-ADL, QMG, MGC, MG-QoL 15R scale, MGFA clinical classification, and MGFA post-intervention state (MGFA PIS) grading scales, as well as self-antibodies, etc. Safety measurement includes vital signs, physical examination, laboratory tests, cytokines, and ECG, etc. The adverse events and concomitant therapy will be continuously collected during the trial. In addition, during the study period, blood samples will be collected from participants who have received SYS6020 treatment for PK/PD test, and immunogenicity test. For the dose-escalation phase, 3 to 5 dose levels are proposed to be explored. The Safety Monitoring Committee (SMC) will discuss the safety data and make a decision if the next SYS6020 injection could be initiated or dose-escalation could be initiated. After the completion of the dose-escalation phase, the recommended doses would be determined for dose-expansion phase. For the dose-expansion phase, further safety and efficacy data will be collected among the participants who will receive the recommended dose of SYS6020 injection.
CONDITIONS
Official 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 between 18 and 65 years old
- Diagnosed with generalized myasthenia gravis classified as MGFA II to IV
- Diagnosed with refractory myasthenia gravis
- QMG score greater than 11 at screening and before apheresis
- Positive for acetylcholine receptor antibody (AChR-Ab) and/or muscle-specific receptor tyrosine kinase (MuSK) antibody at screening
- Stable daily glucocorticoid therapy dose not exceeding 40 mg prednisone or equivalent for at least 4 weeks prior to baseline
- Understands the clinical trial and voluntarily signs informed consent
You will not qualify if you...
- Known allergy, hypersensitivity, or intolerance to SYS6020 or its components including dextran 40
- Major uncontrolled chronic diseases increasing participant risk
- Other autoimmune diseases requiring systemic treatment (stable autoimmune thyroid disease with normal function allowed)
- Severe recurrent or active infection affecting participation
- History of positive HIV, positive HBsAg, or positive HBcAb with measurable HBV-DNA
- History of malignant tumors within 5 years or active tumors (localized or certain thymomas allowed)
- Serious respiratory system disease
- History of serious cardiovascular disease including severe rhythm or conduction abnormalities
- Clinically significant abnormal lab findings (e.g., ALT, AST >3x ULN, Scr >1.5x ULN, INR >1.5x ULN)
- Other clinically significant disease or lab abnormalities, alcohol dependence, or drug abuse
- Current psychotic disorder interfering with adherence
- History of primary immunodeficiency or prior organ or stem cell transplantation
- Recent Grade 2 or higher bleeding or need for long-term anticoagulants
- Previous CAR-T or gene therapy
- Recent intravenous immunoglobulin, plasmapheresis, plasma separation, or hemodialysis within 1 month before apheresis
- Recent use of calcineurin inhibitors, cyclophosphamide, neonatal Fc receptor antagonists, or targeted B-cell biological agents within specified timeframes
- Investigator judgment deeming participant unsuitable for the trial for other reasons
AI-Screening
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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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