Pilot Study Evaluating the Safety and Effectiveness of Anti-CD19BCMA CAR-NK Cell Therapy for Children Aged 5 and Older with Difficult-to-Treat Pediatric Rheumatic Diseases

What this Trial Is About

Researchers are evaluating the safety and effectiveness of anti-CD19BCMA CAR-NK cell injection in children aged 5 years and older with refractory pediatric rheumatic diseases. This early phase 1 pilot study focuses on patients whose conditions have not responded well to standard treatments, aiming to explore this new biological therapys potential benefits and risks. Participants will receive chemotherapy drugs fludarabine and cyclophosphamide on days -5, -4, and -3 before receiving multiple doses of the investigational anti-CD19BCMA CAR-NK cells KN5601 using a dose-escalation approach. This single-arm, open-label study does not include a comparison group, and the treatment phase includes careful dose adjustments. Throughout the study, participants will be closely monitored for dose-limiting toxicities up to 28 days after infusion. Researchers will assess clinical remission and inactive disease status at 3, 6, 9, and 12 months, as well as monitor B cell depletion rates for up to 52 weeks. Safety evaluations, laboratory tests, and clinical assessments will be conducted regularly to track responses and adverse effects during the study period ending in late 2027.

Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA CAR-NK Cell Injection for the Treatment of Refractory Pediatric Rheumatic Diseases