Clinical Study of Hospital-manufactured CD19 CAR-T in Children and Adolescents With Acute Lymphoblastic Leukemia

Study Overview
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Brief Summary
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Chimeric antigen receptor T cells (CAR-T cells) have been developed to treat relapsed and refractory hematological malignancies with promising outcome in patients with very poor prognosis. The purpose of this clinical study is to produce the CD19[cluster of differentiation antigen 19] CAR-T (SNUH-CD19-CAR-T) at the investigational site and to evaluate safety and efficacy of SNUH-CD19-CAR-T in children and adolescent with relapsed/refractory B-cell acute lymphoblastic leukemia.

Official Title
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A Phase Ib, Clinical Trial of Hospital-manufactured CD19 Chimeric Antigen Receptor T Cells (SNUH-CD19-CAR-T) in Children and Adolescents With Relapsed or Refractory CD19 Positive Acute Lymphoblastic Leukemia

Conditions
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B-cell Acute Lymphoblastic Leukemia

Intervention / Treatment
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* Biological: SNUH-CD19-CAR-T


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Relapsed or refractory CD19 Positive Acute Lymphoblastic Leukemia. All subjects must be younger than 26 years old at the time of obtaining informed consent a. 2nd or greater BM[bone marrow] relapse OR b. Any BM relapse after allogeneic SCT[stem cell transplant] and must be ≥ 6 months from SCT at the time of SNUH_CD19_CAR-T infusion OR c .Refractory as defined by not achieving a CR after 2 cycles of a standard chemotherapy regimen or chemorefractory as defined by not achieving a CR after 1 cycle of standard chemotherapy for relapsed leukemia OR d. Ineligible for allogeneic SCT because of: Severe comorbid disease Other contraindications to allogeneic SCT conditioning regimen Lack of suitable donor 2. Documentation of CD19 tumor expression in bone marrow or peripheral blood by flow cytometry. 3. Karnofsky (age ≥ 16 years) or Lansky (age < 16 years) performance status ≥ 50 at screening Exclusion Criteria: Evidence of uncontrolled hepatitis B virus (HBV) or hepatitis C virus (HCV) based on assessment done by treating physicians. Known human immunodeficiency virus (HIV) infection. Presence of clinically active uncontrolled infection based on assessment done by treating physicians. Infections are considered controlled if appropriate therapy has been instituted and, at the time of screening, no signs of progression are present. Persisting fever without other signs or symptoms will not be interpreted as progressing infection. Pregnant or nursing (lactating) women.

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 25 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: CD19 CAR-T therapy<br>SNUH-CD19-CAR-T is administered as an intravenous infusion. | Biological: SNUH-CD19-CAR-T<br>* SNUH-CD19-CAR-T is an autologous CAR-T from T cells collected from each patient. Administer a single dose of SNUH-CD19-CAR-T to patients with relapsed or refractory CD19 positive B-cell acute lymphoblastic leukemia, and evaluate safety and efficacy of SNUH-CD19-CAR-T for 12 months after the infusion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events and its severity |  | 12 months post SNUH-CD19-CAR-T infusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patients with CR[complete remission] after Hospital-manufactured CAR-T infusion |  | 1 month post SNUH-CD19-CAR-T infusion | 
| Overall survival and event-free survival |  | 12 months post SNUH-CD19-CAR-T infusion |