CAR-T cell therapy for chronic or refractory immune thrombocytopenia

An Investigator-initiated Trial to Evaluate the Efficacy and Safety of CAR-T Cells Therapy in the Treatment of Chronic or Refractory Primary Immune Thrombocytopenia (ITP)

This study is testing if CAR-T cell therapy can help people with chronic or hard-to-treat immune thrombocytopenia improve their platelet counts and overall health.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 8 Years to 75 Years
• Sex: All
• Sponsor: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China (other)
• Drugs / interventions: chemotherapy, CAR-T, chimeric antigen receptor, cyclophosphamide, fludarabine
• Locations: 1 site (Kunming, Yunnan)
• Trial ID: NCT06352281 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of CAR-T cell therapy in patients suffering from chronic or refractory primary immune thrombocytopenia (ITP). Participants will undergo a leukapheresis procedure to manufacture CAR-modified T cells, followed by a lymphodepletion therapy with fludarabine and cyclophosphamide. The CAR-T cells will then be infused into the patients, who will be monitored for safety and efficacy for up to 12 weeks, with extended follow-up for those achieving durable remission. The study is designed to assess the potential of this innovative therapy in managing a challenging hematological condition.

Who should consider this trial

Good fit: Ideal candidates include men and women aged 8-75 diagnosed with chronic or refractory ITP who have not responded adequately to standard treatments.

Not a fit: Patients with thrombocytopenia caused by conditions other than ITP or those who do not meet the inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for patients with chronic or refractory ITP, potentially leading to improved platelet counts and reduced bleeding risks.

How similar studies have performed: While CAR-T cell therapy has shown promise in other hematological conditions, this specific application for ITP is relatively novel and has not been extensively tested in prior studies.

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;

Where this trial is running

Kunming, Yunnan

• 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China — Kunming, Yunnan, China (RECRUITING)

Study contacts

• Principal investigator: Sanbin Wang, Doctor — 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
• Study coordinator: Sanbin Wang, Doctor

Conditions: ITP - Immune Thrombocytopenia, CAR-T, Immune Thrombocytopenia