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| Name | Class |
|---|---|
| Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd | INDUSTRY |
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Anti-BCMA CAR T-cells infusion | Experimental | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| autologous anti-BCMA chimeric antigen receptor T cells | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| overall response | 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. | 6-months |
| Measure | Description | Time Frame |
|---|---|---|
| Time to response | Time since infusion of CAR T-cells to the time to achieve the response. | 6-months |
| Duration of response | Period from the achievement of response to the loss of response. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Soochow University | Recruiting | Suzhou | Jiangsu | 215006 | China |
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| 6-months |
| Incidence of adverse events | 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. | 6-months |
| Evaluation of bleeding events | 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)'). | 6-months |
| Evaluation of concomitant therapy | Duration of discontinuation or dose reduction of concomitant treatment,and the degree of decrease of combined treatment from the baseline in patients. | 6-months |
| Evaluation of health-related quality of life | Health-related quality of life will be evaluated according to ITP-PAQ (Primary Immune Thrombocytopenia Patient Assessment Questionnaire). | 6-months |