a Clinical Research of BCMA-Targeted Prime CAR-T Cell Therapy in Relapsed/Refractory Multiple Myeloma

Study Overview
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Brief Summary
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This is a single arm study to evaluate the efficacy and safety of BCMA-targeted prime CAR-T cells therapy for patients with relapsed/refractory Multiple Myeloma.

Detailed Description
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There are limited options for treatment of relapse/refractory Multiple Myeloma. BCMA is expressed on most Multiple Myeloma cells so it is an ideal target for CAR-T. In this study, investigators will evaluate the safety and efficacy of prime CAR- T targeting BCMA in patients with relapsed/refractory Multiple Myeloma. The primary goal is safety and efficiency assessment, including adverse events and disease status after treatment.

Official Title
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Study Evaluating Safety and Efficacy of BCMA-Targeted Prime CAR-T Cell in Patients With Relapsed/Refractory Multiple Myeloma

Conditions
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Multiple Myeloma, Neoplasm, Plasma Cell, Multiple Myeloma in Relapse

Intervention / Treatment
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* Biological: BCMA targeted prime CAR-T cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed written informed consent; Diagnose as relapsed /refractory multiple myeloma, and meet one of the following conditions: Failed to standard chemotherapy regimens; Relapse after complete remission, high-risk and / or refractory patients ; Relapse after hematopoietic stem cell transplantation; Evidence for cell membrane BCMA expression All genders, ages: 18 to 75 years# The expect time of survive is above 3 months; KPS>60 No serious mental disorders ; Left ventricular ejection fraction ≥50% Sufficient hepatic function defined by ALT/AST≤3 x ULN and bilirubin≤2 x ULN; Sufficient renal function defined by creatinine clearance≤2 x ULN; Sufficient pulmonary function defined by indoor oxygen saturation≥92%; 12. With single or venous blood collection standards, and no other cell collection contraindications; 13. Ability and willingness to adhere to the study visit schedule and all protocol requirements. Exclusion Criteria: Previous history of other malignancy; Presence of uncontrolled active infection; Evidence of disorder that need the treatment by glucocorticoids; Active or chronic GVHD The patients treatment by inhibitor of T cell Pregnant or breasting-feeding women; Any situation that investigators regard not suitable for attending in this study (e.g. HIV , HCVinfection or intravenous drug addiction) or may affect the data analysis.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BCMA targeted prime CAR-T cells treat<br>Patients will be be treated with BCMA targeted prime CAR-T cells | Biological: BCMA targeted prime CAR-T cells<br>* BCMA targeted prime CAR-T cell therapy<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events that related to treatment | Therapy-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0) | 2 years | 
| The response rate of BCMA targeted prime CAR-T treatment in patients with relapse/refractory Multiple Myeloma that treatment by BCMA targeted prime CAR-T cells therapy | The response rate of BCMA targeted prime CAR-T treatment will be recorded and assessed according to the IMWG | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of BCMA targeted prime CAR-T cells in bone marrow and peripheral blood | In vivo (bone marrow and peripheral blood) rate of BCMA targeted prime CAR-T cells were determined by means of flow cytometry | 2 years | 
| Quantity of BCMA targeted prime CAR copies in bone marrow and peripheral blood | In vivo (bone marrow and peripheral blood) quantity of BCMA targeted prime CAR copies were determined by means of qPCR | 2 years | 
| Quantity of clonal plasma cells in bone marrow | In vivo (bone marrow) quantity of clonal plasma cells | 1 years | 
| Levels of IL-6 in Serum | In vivo (Serum) quantity of IL-6 | 1years | 
| Duration of Response (DOR) of BCMA targeted prime CAR-T treatment in patients with refractory/relapsed multiple myeloma | DOR will be assessed from the first assessment of sCR/CR/VGPR/PR to the first assessment of recurrence or progression of the disease or death from any cause (censored) | 2 years | 
| Progress-free survival(PFS) of BCMA targeted prime CAR-T treatment in patients with refractory/relapsed multiple myeloma | PFS will be assessed from the first prime CAR-T cell infusion to death from any cause or the first assessment of progression (censored) | 2 years | 
| Overall survival(OS) of BCMA targeted prime CAR-T treatment in patients with refractory/relapsed multiple myeloma | OS will be assessed from the first prime CAR-T cell infusion to death from any cause (censored) | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Multiple Myeloma, BCMA, prime Chimeric Antigen Receptor T Cell