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Anti-BCMA CAR T-Cell Therapy for R/R ITP

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05315778
Recruitment Status : Recruiting
First Posted : April 7, 2022
Last Update Posted : April 7, 2022
Sponsor:
Collaborator:
Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd
Information provided by (Responsible Party):
The First Affiliated Hospital of Soochow University

Study Description
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Brief Summary:
This is a prospective, single-center, open-label, single-arm study, to evaluate the efficacy and safety of Anti-BCMA chimeric antigen receptor T cell therapy（BCMA CAR-T）for patients with relapse/refractory Immune thrombocytopenia(R/R ITP).

Condition or disease Intervention/treatment Phase
ITP Biological: autologous anti-BCMA chimeric antigen receptor T cells Phase 2

Detailed Description:
Immune thrombocytopenia (ITP) is a disorder that can lead to easy or excessive bruising and bleeding. Approximately two-thirds of patients achieve remission after/during first-line therapies. However, the other part of patients could not achieve durable remission or even refractory to initial treatments. Those cases, known as relapse/refractory Immune thrombocytopenia (R/R ITP), undergo the heavy burden of disease which decreases the quality of life. Lots of pathogeneses take part in the occurrence of R/R ITP, and the most important one of them is antibody-mediated immune platelet destruction. As far as it is known，human platelet autoantibodies are mainly secreted by plasma cells, especially long-lived plasma cells. Researchers want to explore that can BCMA CAR-T help R/R ITP patients increase platelet count, reduce bleeding episodes and the dose of concomitant medications.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Anti-BCMA CAR T-Cell Therapy for Relapsed/Refractory Immune Thrombocytopenia
Actual Study Start Date : January 1, 2022
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : June 30, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Anti-BCMA CAR T-cells infusion
R/R ITP patients will accept infusion of autologous anti-BCMA CAR T-cells with a total of 1.0-2.0×10e7/Kg. The patients will be follow-up for 6 months post CAR T-cell therapy.
 Biological: autologous anti-BCMA chimeric antigen receptor T cells
Lymphoadenodepletion chemotherapy with FC (fludarabine 30mg/ m2 for 3 consecutive days and cyclophosphamide 300mg/m2 for 3 consecutive days) will be given at day -5, -4 and -3 before CAR T-cells infusion. A total of 1.0-2.0×10e7/Kg autologous anti-BCMA CAR T-cells will be infused by dose-escalation after the lymphoadenodepletion chemotherapy. Dose of CAR T-cells are allowed to be adjusted according to the severity of cytokine release syndrome.


Outcome Measures
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Primary Outcome Measures :
  1. overall response [ Time Frame: 6-months ]
    The number of participants who achieved CR (defined as platelet count≥100x10e9/L) and PR (defined as platelet count ≥30x10e9/L and at least a 2-fold increase the baseline count and absence of bleeding) at follow-up.


Secondary Outcome Measures :
  1. Time to response [ Time Frame: 6-months ]
    Time since infusion of CAR T-cells to the time to achieve the response.

  2. Duration of response [ Time Frame: 6-months ]
    Period from the achievement of response to the loss of response.

  3. Incidence of adverse events [ Time Frame: 6-months ]
    Adverse events will be assessed daily during the first 2 weeks after the BCMA CAR-T treatment, and monthly thereafter. Adverse events will be evaluated according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

  4. Evaluation of bleeding events [ Time Frame: 6-months ]
    Bleeding events will be evaluated according to Bleeding rating system of ITP('Chinese guideline on the diagnosis and management of adult primary immune thrombocytopenia (version 2020)').

  5. Evaluation of concomitant therapy [ Time Frame: 6-months ]
    Duration of discontinuation or dose reduction of concomitant treatment，and the degree of decrease of combined treatment from the baseline in patients.

  6. Evaluation of health-related quality of life [ Time Frame: 6-months ]
    Health-related quality of life will be evaluated according to ITP-PAQ (Primary Immune Thrombocytopenia Patient Assessment Questionnaire).


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Refractory ITP defined according to the recent consensual criteria ( 'Chinese guideline on the diagnosis and management of adult primary immune thrombocytopenia (version 2020)'), or relapse ITP defined as ITP patients who have responded to first-line therapy (glucocorticoids or immunoglobulins) and anti-CD20 monoclonal antibody, but cannot maintain the response.
  • Ages 18-65 years inclusive.
  • Adequate venous access for apheresis or venous blood and no other contraindications for leukocytosis.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
  • Subjects should have full capacity for civil conduct, understand necessary information，sign the informed consent form voluntarily，and have good corporation with the content of this research protocol.

Exclusion Criteria:

  • Secondary ITP.
  • Patients with a known history or prior diagnosis of arterial thrombosis (such as cerebral thrombosis, myocardial infarction, etc.), or comorbidity of venous thrombosis (such as deep vein thrombosis, pulmonary embolism), or are using anticoagulant/antiplatelet drug at the beginning of trial.
  • Patients with a known history or prior diagnosis of serious cardiovascular disease.
  • Patients with uncontrolled infection, organ dysfunction or any uncontrolled active medical disorder that would preclude participation as outlined.
  • Patients with malignancy or history of malignancy.
  • Failed T cell expansion test.
  • During screening, hemoglobin <100g/L; absolute value of neutrophil count <1.5×10^9/L.
  • During screening, serum creatinine concentration > 1.5x the upper limit of the normal range, total bilirubin > 1.5x the upper limit of the normal range, alanine aminotransferase and aspartate aminotransferase > 3x the upper limit of the normal range, Left ventricular ejection fraction ≤ 50% by echocardiography, Pulmonary function ≥ grade 1 dyspnea (CTCAE v5.0), blood oxygen saturation<91% without oxygen inhalation.
  • Prothrombin time (PT) or prothrombin time-international normalized ratio (PT-INR) or activated partial thromboplastin time (APTT) exceeding 20% of the normal reference range; or a history of coagulation abnormalities other than ITP.
  • Either HIV antibody or syphilis antibody is positive; hepatitis C antibody is positive and the detection of HCV-RNA exceeds the laboratory test upper reference limit; hepatitis B surface antigen is positive and the detection of HBV-DNA exceeds the laboratory test upper reference limit.
  • Participated in other clinical studies within 3 months before this CAR-T cell infusion.
  • Patients is pregnant or breastfeeding, or planning pregnancy.
  • Patients is fertile and the investigator determines the case is inappropriate to participate.
  • History of severe drug allergy or known allergy to CAR-T treatment related drugs.
  • Suspected or established alcohol, drug or drug abuse.
  • The investigator judges that it is not suitable to participate in this trial.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05315778


Locations
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China, Jiangsu
The First Affiliated Hospital of Soochow University Recruiting
Suzhou, Jiangsu, China, 215006
Contact: Xiaowen Tang, Ph.D    86-512677801856    xwtang1020@163.com   
Sponsors and Collaborators
The First Affiliated Hospital of Soochow University
Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd
More Information
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Responsible Party: The First Affiliated Hospital of Soochow University
ClinicalTrials.gov Identifier: NCT05315778    
Other Study ID Numbers: BCRITP
First Posted: April 7, 2022    Key Record Dates
Last Update Posted: April 7, 2022
Last Verified: March 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by The First Affiliated Hospital of Soochow University:
CAR-T
BCMA