Efficacy and Safety of CAR-T Cells Therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP)

The 920th Hospital of The Joint Logistics Support Force of the Chinese People's Liberation Army

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

ITP - Immune Thrombocytopenia

Treatments

Biological: CAR-T cells

Study type

Interventional

Funder types

Other

Identifiers

NCT06352281

KM-007

Details and patient eligibility

About

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).

Full description

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.

Enrollment

10 estimated patients

Sex

All

Ages

8 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Willingness to complete the informed consent process and to comply with study procedures and visit schedule;
Men and women aged 8-75;
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;
The results of physical, instrumental, and laboratory examination of patients not suggest any disease which may cause thrombocytopenia other than ITP;
Platelet count <30 x 109 / L;
If the patient is taking corticosteroids, the treatment regimen/dose should be stable (at least 2 weeks prior to screening);
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;
Willingness to use effective and reliable methods of contraception throughout the entire study period;

Exclusion criteria

All subjects with diseases which may cause secondary immune thrombocytopenia
Patients with preventive splenectomy;
Hemostatic disorders other than chronic thrombocytopenia;
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;
History of platelet agglutination abnormality that prevents reliable measurement of platelet counts;
Concurrent malignant disease and/or history of cancer treatment with cytotoxic chemotherapy and/or radiotherapy;
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;
History of thrombosis or presence of significant risk factors for thrombosis;
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;
Any clinically significant hepatic impairment (increase of serum transaminase levels by more than 3 times the upper limit of normal);
Serum creatinine levels are more than two times higher than the upper limit of normal for a given age and sex;
Any other concomitant decompensated diseases or acute conditions, the presence of which, according to the researcher, may significantly affect the results of the study;
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;
Patients with severe history of allergy or allergic constitution;
Pregnancy and lactation;
History of mental illness and known alcohol/drug addiction;
Poor compliance due to physiological, family, social, geographical and other factors, unable to cooperate with the study protocol and follow-up plan;
Had undergone other clinical trials in the 4 weeks prior to participating in this trial;