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

NCT06688435 · tracked via the Priya Life Science China tracker

Sponsor

CSPC ZhongQi Pharmaceutical Technology Co., Ltd

Phase

Phase 1

Started

2025-03-04

Last updated

2026-08-12

Condition(s) studied

Myasthenia Gravis

Investigational drug(s) / intervention(s)

SYS6020 injection →SYS6020 injection →SYS6020 injection →

SYS6020 injection: The SYS6020 is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. Before each dosing, the infusion eligibility of SYS6020 is assessed by the investigator, and the eligible participants will receive 3 dosing SYS6020 injection treatment. The dosing interval is 7 days according to the protocol design.

SYS6020 injection: The SYS6020 is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. Before each dosing, the infusion eligibility of SYS6020 is assessed by the investigator. The eligible participants will receive SYS6020 injection treatment twice weekly for 3 weeks, for a total of 6 doses.

SYS6020 injection: The SYS6020 is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. Before each dosing, the infusion eligibility of SYS6020 is assessed by the investigator. The eligible participants will receive SYS6020 injection treatment once weekly for 6 weeks, for a total of 6 doses.

Study summary

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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

65 Years

Healthy volunteers

Inclusion Criteria: * 1\) The ages ≥18 and ≤ 70 years old; * 2\) Diagnosed as generalized myasthenia gravis (GMG), the clinical classification of MGFA II-IVa; * 3\) Diagnosed as refractory myasthenia gravis (refractory MG) ； * 4\) QMG score \>11 in the screening period and before apheresis; * 5\) Positive acetylcholine receptor antibody (AChR-Ab) and/or muscle-specific receptor tyrosine kinase (MuSK) antibody in the screening period; * 6\) The daily dose of concomitant glucocorticoid therapy must not exceed 20mg prednisone or equivalent within 7 days prior to apheresis; * 7\) Participants have a thorough understanding of this clinical trial and voluntarily sign a written informed consent form. Exclusion Criteria: * 1\) Have been known to have allergic reactions, hypersensitivity, intolerance or contraindications to SYS6020（including its active ingredient and excipient dextran 40） or the drugs potentially used in the study, or who have had a previous history of severe allergic reactions; * 2\) Participants with major chronic diseases that are not well-controlled and considered to increase the participant's risk potentially by the investigator; * 3\) Participants with other autoimmune diseases that require systemic treatment. Participants with stable autoimmune thyroid diseases who have a normal thyroid function and are at a stable therapeutic dose are allowed to be enrolled. * 4\) Participants with a severe recurrent infection during the screening period, or any active infection that the investigator considers may affect the patient's participation; * 5)Participants with a history of positive HIV; participants with positive HBsAg; participants with positive HBcAb and with HBV-DNA above the measurable limit;(Note: participants with positive HBV-DNA or HCV-RNA results within 6 months prior to ICF are excluded); * 6\) Participants with a history of malignant tumors within the past 5 years or with current active malignant tumors. (Participants with successfully treated localized tumors, as well as participants with WHO histological type A, AB, B1 or B2thymoma, with no evidence of recurrence or metastasis for at least 1 year after complete resection assessed by the investigator , are allowed to be enrolled;) * 7\) Any serious respiratory system disease. * 8\) Participants with a history of serious cardiovascular disease, such as severe cardiac rhythm or conduction abnormalities. * 9\) Abnormal laboratory findings with clinical significance, including ALT, AST\>3\*ULN； Scr\>1.5\*ULN； INR\>1.5\*ULN, and so on. . * 10\) Individuals with potential disease conditions (including laboratory abnormalities) which are considered of clinical significance by the investigator; individuals with alcohol dependence or drug abuse . * 11\) Individuals with a current psychotic disorder that interferes with adherence. * 12\) Participants with a history of primary immunodeficiency disease, organ or hematopoietic stem cell/bone marrow transplantations before screening; or those planning to undergo a transplantation during the trial; * 13\) Participants with a history of ≥ Grade 2 (CTCAE 5.0 standard) bleeding within 30 days before screening, or those requiring long-term continuous treatments with anticoagulant drugs. * 14\) Participants who have received any CAR-T therapy or gene therapy before. * 15\) Participants who have received intravenous injection of human immunoglobulin (IVIG) or plasmapheresis (PE), plasma separation, or hemodialysis within 1 month before apheresis. * 16\) Participants who have used calcineurin inhibitors, or cyclophosphamide or neonatal Fc receptor antagonists within 3 weeks before apheresis. Participants who have used targeted B-cell biological agents such as rituximab within 3 months before apheresis. Participants who started receiving eculizumab treatment within 8 weeks before the first dosing； * 17\) Any situations that the investigator believes that the participant is not suitable for this clinical trial for any other reasons.

Primary outcome measure(s)

The frequency and the grade of DLT and the incidence of adverse events and serious adverse events. (For the dose-escalation phase) — 12 months
For the dose-escalation phase, the primary objective is to evaluate the safety of SYS6020, as measured by the frequency and nature of Dose-limiting toxicity (DLT) and the incidence of all adverse events and serious adverse events. DLT is defined as any adverse event related to SYS6020 that occurs within 28 days after the infusion of SYS6020 and that meets certain criteria.
Response rate at 6 months after dosing （For the dose-expansion phase) — 6months
Response is defined as a ≥3-point reduction from baseline in the MG-ADL total score at Month 6

Trial sites (1)

Facility	City	Region	Status
Qilu Hospital, Shandong University	Jinan	China	Recruiting

More CSPC ZhongQi Pharmaceutical Technology Co., Ltd. trials in China

Evaluation of Bioequivalence and Food Effect of a New Strength Formulation of Ammoxetine Hydrochloride Enteric-coated Tablets in Healthy Participants. Recruiting

SYH2095 for Advanced Malignant Tumors: Phase I Study Starting soon

Paclitaxel Cationic Liposome (Hepatic Arterial Infusion) in Combination With Systemic Therapy as First-Line Treatment in Colorectal Liver Metastases Recruiting

A Study of Sirolimus (Albumin-Bound) in Combination With Different ADCs Treatment of Advanced Solid Tumors Recruiting

Sirolimus for Injection (Albumin-bound) in Combination With Endocrine Therapy for HR+/HER2- Advanced/Metastatic Breast Cancer Patients Who Have Failed Standard Therapy Active, not recruiting

Other trials for the same condition

Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/ BCMA（QT-019C） in Patients With r/ r Neurological Autoimmune Diseases Starting soon

Precision Diagnosis and Prospective Cohort Study for Myasthenia Gravis: Multicenter Analysis in China Recruiting

Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis Recruiting

A Study in Patients With Myasthenia Gravis in China Active, not recruiting

Cohort Study on Neuroimmune Diseases in the Reproductive Age Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06688435 on ClinicalTrials.gov ↗ ← All trials in China