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)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China1 site in 1 country10 target enrollmentFebruary 1, 2024

ConditionsITP - Immune Thrombocytopenia

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: ITP - Immune Thrombocytopenia
Sponsor: 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Enrollment: 10
Locations: 1
Primary Endpoint: Portion of patients with response (R)
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

February 1, 2024

End Date

December 31, 2027

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Responsible Party

Sponsor

Eligibility Criteria

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);

Outcomes

Primary Outcomes

Portion of patients with response (R)

Time Frame: At least 2 weeks after infusion

platelet count\>30x10\^9/L and at least 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding

Secondary Outcomes

Portion of patients with complete response (CR)(At least 2 weeks after infusion)
Time (in days) from treatment start to response (R)(At least 2 weeks after infusion)
Duration (in days) of response (R)(At least 2 weeks after infusion)
Incidence of adverse events(AE) after infusion(Up to 12 months after infusion)
Time (in days) from treatment to complete response (CR)(At least 2 weeks after infusion)
Portion of patients with relapse(At least 2 weeks after infusion)