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
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|ITP||Biological: autologous anti-BCMA chimeric antigen receptor T cells||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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.
- 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.
- Time to response [ Time Frame: 6-months ]Time since infusion of CAR T-cells to the time to achieve the response.
- Duration of response [ Time Frame: 6-months ]Period from the achievement of response to the loss of response.
- 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.
- 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)').
- 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.
- 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).
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years to 65 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
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
|The First Affiliated Hospital of Soochow University||Recruiting|
|Suzhou, Jiangsu, China, 215006|
|Contact: Xiaowen Tang, Ph.D 86-512677801856 firstname.lastname@example.org|
|Responsible Party:||The First Affiliated Hospital of Soochow University|
|Other Study ID Numbers:||
|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|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|