Anti CD19/BCMA CAR Gene Therapy for Relapsed/Refractory Immune Thrombocytopenia

This dose-escalation study evaluates the safety and effectiveness of an anti-CD19/BCMA CAR gene therapy — a type of cell therapy that reprograms immune cells to attack the immune cells causing the disease — in adults with immune thrombocytopenia (ITP) that has not responded to multiple prior treatments. ITP is a condition where the immune system mistakenly destroys platelets, leading to dangerous bleeding, and this gene therapy approach targets the root immune dysfunction. Eligible participants are adults 18 and older with primary ITP for at least 6 months, platelet counts below 30×10⁹/L, positive anti-platelet antibodies, and documented failure of both first- and second-line therapies including TPO agonists and/or rituximab; those with heart failure, active infections, or prior serious cardiac events are excluded. Participation involves receiving the CAR gene therapy infusion along with blood tests and monitoring for safety and platelet response over the course of the study. This summary was prepared using AI to help patients understand the study in plain language.