Study of Anti-CD19BCMA CAR Gene Therapy for Adults with Relapsed or Refractory Immune Thrombocytopenia

What this Trial Is About

Researchers are studying an investigational gene therapy called Anti-CD19BCMA CAR gene vector injection for adults with relapsed and refractory immune thrombocytopenia ITP, a condition where the immune system attacks platelets. This open-label, single-site, dose-escalation study aims to assess the safety and efficacy of this treatment in up to 18 participants. The research is sponsored by Anhui Provincial Hospital and monitors outcomes over a one-year period. Participants receive a single intravenous infusion of the Anti-CD19BCMA CAR gene vector, with doses ranging from 1.0 to 4.0 million transducing units per kilogram. This experimental treatment is given once, and the study does not include placebo or comparison groups. The study focuses on how well the gene vector persists and the overall remission rate following treatment, alongside monitoring for adverse events. During the study, participants will undergo safety assessments, including tracking adverse events for up to one year. Researchers will monitor the persistence of the gene therapy in the body and evaluate the remission of ITP symptoms. Participants must agree to contraception requirements and will have regular clinical evaluations and laboratory tests to assess organ function and blood counts. The total study duration spans up to one year after treatment to ensure thorough safety and efficacy evaluation.

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