Study of Anti-BCMACD70 CAR-T Cell Therapy for Patients 2 Years and Older with Multi-Drug Resistant Steroid-Resistant Nephrotic Syndrome

What this Trial Is About

Researchers are evaluating the safety and effectiveness of anti-BCMACD70 CAR-T cell therapy in patients with Multi-Drug Resistant Steroid-Resistant Nephrotic Syndrome MDR-SRNS, a condition that currently lacks effective treatments and carries a high risk of progressing to kidney failure. This trial aims to address the urgent need for new therapies, as many patients experience disease recurrence even after kidney transplantation. CAR-T therapy has shown promise in treating autoimmune diseases, suggesting potential benefits for MDR-SRNS. The study includes two phases a Dose Exploration phase Part A and a Dose Expansion phase Part B. Part A tests three escalating doses of anti-BCMACD70 CAR-T cells 0.3105kg, 1105kg, 3105kg to find a safe and effective dose, with the possibility to increase the dose up to 6105kg if needed. After determining the optimal dose, Part B will enroll additional patients to further assess safety and efficacy. A total of 12 to 18 patients are expected to participate. Participants will be closely monitored through clinical evaluations over several months, with safety assessed at 3 months and treatment effectiveness at 6 months. The study includes assessments of cellular kinetics and organ function. Patients or their guardians will provide informed consent and follow specific procedures, including blood tests and organ function evaluations, to support treatment and safety monitoring throughout the trial period.

Study of Therapeutic Efficacy of CAR-T Cell Therapy in Patients With MDR-SRNS