Phase 1 Study of CD19-Targeted CAR-T Therapy for Children and Teens with Difficult-to-Treat Juvenile Dermatomyositis

What this Trial Is About

This research aims to evaluate the safety and effectiveness of CD19-targeted CAR-T therapy in treating refractory juvenile dermatomyositis RJDM, a chronic autoimmune disease affecting children that is often resistant to standard treatments. The study is a Phase I trial sponsored by Chongqing Precision Biotech Co., Ltd and involves children aged 5 to 17 years. RJDM patients often experience severe symptoms and require long-term use of hormones and immunosuppressants, so this trial explores a new cellular therapy approach. The trial is divided into two parts dose exploration Part A and dose extension Part B. Participants will first undergo chemotherapy to clear lymphocytes, followed by a single intravenous infusion of CD19-targeted CAR-T cells. This therapy targets B cells that contribute to the autoimmune condition, aiming to rebuild the immune system. The study will monitor how the cells behave in the body over time and assess the appropriate dosing. Participants will be closely followed for safety and treatment response, with evaluations at 28 days, 2 months, 3 months, and up to 2 years to assess safety, effectiveness, and duration of response. Researchers will collect data on how the CAR-T cells circulate and persist in the body. Organ functions, antibody levels, muscle strength, skin symptoms, and other clinical signs will be monitored. The total study duration allows for long-term observation of treatment effects and potential side effects.

Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis