CD38-targeted Chimeric Antigen Receptor T Cell (CART) in Relapesd or Refractory Acute Myeloid Leukemia

Study Overview
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Brief Summary
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This is a single center, open-label phase 1/2 study to evaluate the safety and efficacy of targeted CD38 chimeric antigen receptor engineered T cell immunotherapy (CART) in the treatment of CD38 positive relapsed or refractory acute myeloid leukemia.

Detailed Description
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The patients will receive infusion of CAR T-cells targeting CD38 to confirm the safety and efficacy of CD38 CAR T-Cells in relapsed or refractory acute myeloid leukemia.

Official Title
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Pilot Study of the Efficacy and Safety of CD38 Targeted Chimeric Antigen Receptor Engineered T-Cells in the Treatment of CD38 Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

Conditions
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Acute Myeloid Leukemia

Intervention / Treatment
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* Biological: CART-38


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: CD38 positive relapsed/refractory acute myeloid leukemia age 6-65 years. left ventricular ejection fractions ≥ 0.5 by echocardiography creatinine < 1.6 mg/dL aspartate aminotransferase/aspartate aminotransferase < 3x upper limit of normal Total bilirubin <2.0 mg/dL karnofsky performance status ≥ 60 expected survival time ≥ 3 months (according to investigator's judgement) Exclusion Criteria: patients are pregnant or lactating uncontrolled active infection class III/IV cardiovascular disability according to the New York Heart Association Classification active hepatitis B or hepatitis C infection patients with HIV infection patients with history of seizure active central nervous system leukemia

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 65 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: CD38 positive relapsed or refractory acute myeloid leukemia<br> | Biological: CART-38<br>* Split intravenous infusion of CART-38 cells [dose escalating infusion of (5-20)x10^6 CART-38 cells/kg].<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Adverse Events | Adverse events are evaluated with CTCAE V5.0 | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response rate (ORR) | ORR includes CR, CRi, MLFS and PR. Complete remission (CR)：Bone marrow blasts <5%; absence of circulating blasts and blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count >1.0x 10^9/L; platelet count >100x10^9/L. CR with incomplete hematologic recovery (CRi)：All CR criteria except for residual neutropenia (<1.0x10^9/L) or thrombocytopenia (<100x10^9/L). Morphologic leukemia-free state (MLFS): Bone marrow blasts <5%; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required. Partial remission (PR): All hematologic criteria of CR; decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%. | 2 years | 
| Event-free survival (EFS) | time from enrollment to the date of primary refractory disease, or relapse from CR or CRi, or death from any cause. | 2 years | 
| Cumulative incidence of relapse(CIR) | time from the date of achievement of a remission until the date of relapse. | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CD38, CART, AML