Abstract: TH-OR090
Safety and Efficacy of B Cell Maturation Antigen (BCMA)/CD70-Targeted Chimeric Antigen Receptor T Cell (CAR-T) Therapy in Pediatric Patients with Multidrug-Resistant, Steroid-Resistant Nephrotic Syndrome
Session Information
- Late-Breaking Research Orals - 1
November 06, 2025 | Location: Grand Ballroom C, Convention Center
Abstract Time: 05:42 PM - 05:54 PM
Category: Pediatric Nephrology
- 1900 Pediatric Nephrology
Authors
- Meng, Hanyan, Children's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Huang, Guoping, Children's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Liu, Fei, Children's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Zhang, Yingzi, Chongqing Precision Biotech Co., Ltd., Chongqing, China
- He, Xue, Children's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Wang, Jingjing, Children's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
- Mao, Jianhua, Children's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China
Background
Patients with SRNS who could not achieve CR after 12 months of treatment with 2 mechanistically distinct steroid-sparing agents administered at standard doses are defined as MDR-SRNS. Currently, available treatments for MDR-SRNS are limited. Here we discuss the safety and efficacy of BCMA/CD70-targeted CAR T-Cell therapy in pediatric patients with MDR-SRNS.
Methods
In this Phase I/II study (NCT06553898), C-4-29 CAR T cells were manufactured by the PRIMCAR manufacturing platform. Per protocol, this study employed a dose-escalation and expansion design. Toxicity was graded by CTCAE V5.0, CRS and ICANS were graded by ASTCT criteria.
Results
By August 2025, 8 patients were infused with C-4-29 (Table 1). 4 experienced CRS, all at grade 1. No ICANS were observed. Among the 8 patients, 1 could not be evaluated for treatment efficacy due to the short duration since reinfusion. 4 achieved CR, and 2 attained PR. However, as of the latest assessment, 5 exhibited an increase in urinary protein excretion, suggesting disease relapse.
Conclusion
BCMA/CD70-targeted CAR T-Cell therapy demonstrates favorable safety profile in pediatric patients with MDR-SRNS while shedding light on disease pathogenesis.The expected completion time of this research is July 2027.
Disease characteristics and safety outcomes
| ID | Age,years | Gender | Disease duration, (months) | Immunosuppressant agents | Kidney biopsy | Dose | CRS | ICANS |
| 1 | 7 | Male | 46 | 6 | MCD | 0.3×10^5/kg | 1 | / |
| 2 | 12 | Female | 127 | 6 | MCD | 0.3×10^5/kg | / | / |
| 3 | 14 | Male | 59 | 4 | FSGS | 0.3×10^5/kg | 1 | / |
| 4 | 8 | Male | 51 | 4 | FSGS | 1.0×10^5/kg | 1 | / |
| 5 | 13 | Female | 44 | 4 | FSGS | 1.0×10^5/kg | / | / |
| 6 | 7 | Female | 45 | 5 | FSGS | 1.0×10^5/kg | 1 | / |
| 7 | 16 | Male | 164 | 5 | FSGS | 1.0×10^5/kg | / | / |
| 9 | 8 | Female | 29 | 6 | FSGS | 1.0×10^5/kg | / | / |