An Exploratory Clinical Study on the Safety and Efficacy of Allogeneic CD19/BCMA CAR-T Cell Treatment for Relapsed/ Refractory B-cell or Plasma Cell-derived Malignant Tumors

A single arm, open-label pilot study is designed to determine the safety and efficacy of CD19 and B-cell maturation antigen (BCMA) targeted allogenic CAR-T cells (RN1101) in patients with relapsed/refractory B-cell or plasma cell-derived malignant tumors. 21 patients are planned to be enrolled in the dose-escalation trial. The primary objective of the study is to evaluation of the safety and feasibility of RN1101 for the treatment of relapsed/refractory B-cell or plasma cell-derived malignant tumors. The secondary objective is to evaluate the efficacy of RN1101 for the treatment of relapsed/refractory B-cell or plasma cell-derived malignant tumors. The exploratory objective is to evaluate expansion, persistence and ability of RN1101 to deplete CD19 or BCMA positive cells in patients with relapsed/refractory B-cell or plasma cell-derived malignant tumors.

开始日期2025-05-06

申办/合作机构

江苏大学附属医院

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100 项与 RN1101 相关的临床结果

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100 项与 RN1101 相关的转化医学

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100 项与 RN1101 相关的专利（医药）

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项与 RN1101 相关的文献（医药）

2026-02-01·Med

Single-cell profiling of immune reset in patients with refractory generalized myasthenia gravis receiving autologous CD19/BCMA CAR-T cell therapy

Article

作者: Li, Shuang ; Li, Hang ; Song, Na ; Tang, Yonglan ; Yang, Naibo ; Liu, Qiang ; Li, Ying ; Ning, Fan ; Huang, Xiaoxi ; Cao, Xiangqi ; Ruan, Zhe ; Paul, Friedemann ; Luan, Xiao ; Zhang, Wenyan ; Li, Zhuyi ; Chang, Ting ; Su, Yue

BACKGROUND:

Chimeric antigen receptor (CAR) T cells targeting CD19 or B cell maturation antigen (BCMA) hold great promise to treat neuroimmune disorders, but the efficacy of CD19/BCMA dual-targeting CAR-T cells and their impact on systemic immunity are poorly understood.

METHODS:

In this phase 1 study (ClinicalTrials.gov: NCT06371040), patients with refractory generalized myasthenia gravis (gMG) received autologous CD19/BCMA CAR-T cells without prior lymphodepletion. The primary endpoint was the frequency and severity of treatment-emergent adverse events at week 4. Secondary endpoints included changes in MG-specific scale scores. Single-cell RNA sequencing and flow cytometry were performed to characterize longitudinal changes in B cell, plasma cell, and T cell compartments following CAR-T cell infusion.

FINDINGS:

CD19/BCMA CAR-T cells expanded in vivo, leading to depletion of B cells and plasma cells. All six patients had a favorable safety profile. Minimal symptom expression (MG Activities of Daily Living Score [MG-ADL] = 0) was achieved in five patients by day 90, with responses sustained through day 120-150, and all patients discontinued glucocorticoids while reducing immunosuppressant use. Immune profiling revealed a transient decline in memory B cells and plasma cells. The repopulated B cells exhibited attenuated B cell receptor signaling and increased inhibitory signals derived from bone marrow niche cells. Further, clonal expansion of T and B cells was significantly reduced.

CONCLUSIONS:

CD19/BCMA CAR-T cell therapy is safe and effective in refractory gMG without lymphodepletion, leading to systemic immune reset that warrants future investigations in larger clinical trials.

FUNDING:

This study was supported by the National Key Research and Development Program and the National Natural Science Foundation of China.

100 项与 RN1101 相关的药物交易

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