Phase 1 Study of CD19-Targeted Universal CAR-T Cell Therapy for Treating Refractory Juvenile Dermatomyositis

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a universal CAR-T cell therapy targeting CD19 in treating patients with refractory juvenile dermatomyositis RJDM. This Phase 1, open-label, single-arm dose-escalation trial also aims to study how the therapy behaves in the body, its role in immune system recovery, and potential long-term survival benefits. The study uses a 33 dose-escalation design with three dosing levels 1 107kg, 3 107kg, and 6 107kg. Patients receive a single intravenous infusion of anti-CD19 UCAR-T cells after lymphodepletion chemotherapy. Dose escalation to the next group occurs only if no dose-limiting toxicity is seen. Each dose group will enroll 3 to 6 patients, totaling 9 to 18 participants. Participants will be monitored for adverse events within 28 days and assessed over six months for disease activity, muscle inflammation by MRI, antibody and muscle enzyme changes, and physical function using the Childhood Myositis Assessment Scale. Pharmacokinetics of the infused cells will be tracked over one month, and muscle enzyme changes will be followed for one year. The study continues until long-term safety and efficacy information is collected.

Clinical Study of CD19 Targeted Universal Chimeric Antigen Receptor T Lymphocytes (UCAR-T) for the Treatment of Refractory Juvenile Dermatomyositis (RJDM)