Study of Universal CD7 CAR T Cells RD13-02 for Adults With Relapsed or Refractory Severe Aplastic Anemia Evaluating Safety, Effectiveness, and Drug Behavior in a Phase 1 Trial

What this Trial Is About

Researchers are evaluating RD13-02, a universal CD7 CAR T-cell therapy, in adults with relapsed or refractory severe aplastic anemia SAA. This early-phase study aims to explore the safety, tolerability, how the drug moves and acts in the body, and its effectiveness in this condition. The trial is sponsored by the Institute of Hematology & Blood Diseases Hospital in China and focuses on patients who have not responded to standard treatments. The trial uses a single-arm, open-label design with a dose escalation phase followed by an expansion phase. Each phase includes a screening period of up to 2 weeks, a 7-day conditioning treatment with chemotherapy, then a 28-day treatment period where participants receive a single intravenous infusion of the RD13-02 CAR T cells. The CAR T cells are genetically modified from healthy donors to target CD7 on affected cells while minimizing risks like graft-versus-host disease. Continuous heart monitoring occurs during and after the infusion. Participants will be closely monitored for up to 6 months after the infusion. Regular assessments include safety checks for side effects or toxicities, and measurements of blood cell response at 3 and 6 months. The study involves physical exams, laboratory tests, and ongoing observation to evaluate how well the treatment is tolerated and how it affects the disease. The total study duration includes screening, treatment, and follow-up phases.

CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia