efficacy and safety of car-t cells therapy for chronic or refractory primary immune thrombocytopenia (itp)

Conditions: ITP - Immune Thrombocytopenia

Summary: it is a single-center, single-arm, open-labeled clinical trial to evaluate the efficacy and safety of car-t cells therapy for chronic or refractory primary immune thrombocytopenia (itp).

this open label and single-arm study aims to evaluate the efficacy and safety of car-t cells therapy in patients with chronic or refractory primary immune thrombocytopenia (itp). after enrollment, a leukapheresis procedure will be performed to manufacture chimeric antigen receptor (car) modified t cells. patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide, then the car-t cells will be infused by vein. after infusion, subjects will be followed for safety and efficacy evaluation up to 12 weeks. for those with a durable remission 12 weeks after infusion, the follow-up will last for at least 12 months for disease control.

Eligibility criteria:
inclusion criteria: 1. willingness to complete the informed consent process and to comply with study procedures and visit schedule; 2. men and women aged 8-75; 3. participants diagnosed with chronic (\>12 months duration) or refractory (a documented intolerance or insufficient response to the first and second line standard treatment of itp) itp; 4. the results of physical, instrumental, and laboratory examination of patients not suggest any disease which may cause thrombocytopenia other than itp; 5. platelet count \<30 x 109 / l; 6. if the patient is taking corticosteroids, the treatment regimen/dose should be stable (at least 2 weeks prior to screening); 7. the results of physical, instrumental, and laboratory examination of patients should be within the normal range or deviations should be regarded by the researcher as clinically insignificant; 8. willingness to use effective and reliable methods of contraception throughout the entire study period; exclusion criteria: 1. all subjects with diseases which may cause secondary immune thrombocytopenia 2. patients with preventive splenectomy; 3. hemostatic disorders other than chronic thrombocytopenia; 4. subject treated with drugs that affect platelet function (including but not limited to aspirin, clopidogrel and/or nsaids) or anti-coagulants for \> 3 consecutive days within 2 weeks of the study start and until the end of the study; 5. history of platelet agglutination abnormality that prevents reliable measurement of platelet counts; 6. concurrent malignant disease and/or history of cancer treatment with cytotoxic chemotherapy and/or radiotherapy; 7. grade iii-iv heart failure or myocardial infarction, cardiac angioplasty or stenting, unstable angina pectoris, or other clinically prominent heart disease within one year prior to enrollment; 8. history of thrombosis or presence of significant risk factors for thrombosis; 9. persons with acute or exacerbation of chronic diseases of the gastrointestinal tract associated with the risk of bleeding, acute infectious diseases, pathologies of the respiratory system; 10. any clinically significant hepatic impairment (increase of serum transaminase levels by more than 3 times the upper limit of normal); 11. serum creatinine levels are more than two times higher than the upper limit of normal for a given age and sex; 12. any other concomitant decompensated diseases or acute conditions, the presence of which, according to the researcher, may significantly affect the results of the study; 13. human immunodeficiency virus (hiv) seropositivity, hepatitis b surface antigen positive or hepatitis b core antibody positive and hbv-dna positive, patients with hepatitis c (hcv-rna quantitative test results positive), or the presence of other serious active viral or bacterial infections or uncontrolled systemic fungal infections; 14. patients with severe history of allergy or allergic constitution; 15. pregnancy and lactation; 16. history of mental illness and known alcohol/drug addiction; 17. poor compliance due to physiological, family, social, geographical and other factors, unable to cooperate with the study protocol and follow-up plan; 18. had undergone other clinical trials in the 4 weeks prior to participating in this trial;

Keywords: car-t immune thrombocytopenia (itp)