This is a phase 1/2 clinical trial conducted at a single centre in China investigating the effectiveness and safety of CAR T-cell therapy for chronic or refractory ITP. CAR T-cell therapy in a treatment in which a patient’s own immune cells (specifically T-cells) are re-designed to attack a particular type of unhealthy cell in the body. Participants who can enrol are those aged 8-75 who has primary chronic ITP, defined as having the condition for >12 months, or with refractory ITP, defined as intolerance or ineffective response to first- and second-line ITP therapies. Participants must have a platelet count <30,000 and willing to use reliable methods of contraception for the study duration. Those who are unable to participate in this study include those who may have secondary ITP (in other words another condition that may be causing their ITP), have other blood disorders or cancer, heart failure, liver or kidney failure, history of blood clots, are pregnant, or taking medications that can interfere with platelet function (aspirin, anti-coagulants). Participants must also not have participated in another clinical trial within the 4 weeks prior to beginning this trial. If recruited, participants will receive 3-5 days of fludarabine and cyclophosphamide, medications to deplete white blood cells, which is required prior to initiating CAR T-cell therapy. The study will assess the proportion of patients who have a platelet “response” to this therapy, defined as a platelet count >30,000 and at least 2x increase from baseline without bleeding.
China
Actively Recruiting