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

NCT06352281 | Recruiting Phase 1

• 1 other identifier: KM-007
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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).

Conditions

ITP - Immune Thrombocytopenia

Keywords

CAR-T; Immune Thrombocytopenia (ITP)

Outcome Measures

Primary Outcomes (1)

• Portion of patients with response (R)

  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

  At least 2 weeks after infusion

Secondary Outcomes (6)

• Portion of patients with complete response (CR)

  At least 2 weeks after infusion

• Portion of patients with relapse

  At least 2 weeks after infusion

• Time (in days) from treatment start to response (R)

  At least 2 weeks after infusion

• Time (in days) from treatment to complete response (CR)

  At least 2 weeks after infusion

• Duration (in days) of response (R)

  At least 2 weeks after infusion

Study Arms (1)

CAR-T Cells Therapy

EXPERIMENTAL

Subjects who meet the enrollment conditions will receive intravenous infusion of CAR-T cells after lymphodepletion.

Biological: CAR-T cells

Interventions

CAR-T cells BIOLOGICAL

CAR-T cells will be administered by vein. Before CAR-T infusion，patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide.

CAR-T Cells Therapy

Eligibility Criteria

• Age: 8 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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;

Sponsors & Collaborators

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

Study Sites (1)

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

Kunming, Yunnan, 650000, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Immunotherapy, Adoptive

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Adoptive Transfer; Immunization, Passive; Immunization; Immunotherapy; Immunomodulation; Biological Therapy; Therapeutics; Immunologic Techniques; Investigative Techniques

Study Officials

• Sanbin Wang, Doctor

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sanbin Wang, Doctor

CONTACT

13187424131; sanbin1011@163.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2024
• First Posted: April 8, 2024
• Study Start: February 1, 2024
• Primary Completion: January 1, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: July 22, 2024

Record last verified: 2024-07

Locations

CN (1)