Clinical trial NCT06352281: Efficacy and Safety of CAR-T Cells Therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: ITP - Immune Thrombocytopenia
Keywords: CAR-T
Immune Thrombocytopenia (ITP)
Lead sponsor: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Sponsor class: OTHER
First posted: 2024-04-08
Enrollment: 10
Sex: ALL
Minimum age (years): 8
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).

Detailed 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.

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;

Primary outcomes:
measure: Portion of patients with response (R)
timeFrame: At least 2 weeks after infusion
Secondary outcomes:
measure: Portion of patients with complete response (CR)
timeFrame: At least 2 weeks after infusion
measure: Portion of patients with relapse
timeFrame: At least 2 weeks after infusion
measure: Time (in days) from treatment start to response (R)
timeFrame: At least 2 weeks after infusion
measure: Time (in days) from treatment to complete response (CR)
timeFrame: At least 2 weeks after infusion
measure: Duration (in days) of response (R)
timeFrame: At least 2 weeks after infusion
measure: Incidence of adverse events(AE) after infusion
timeFrame: Up to 12 months after infusion
Other outcomes:
Locations:
city: Kunming
country: China
facility: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
state: Yunnan
Study officials:
affiliation: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
name: Sanbin Wang, Doctor
role: PRINCIPAL_INVESTIGATOR
References: