A Clinical Study Evaluating the Safety and Efficacy of BinD19 Treatment in Childhood R/R ALL and Lymphoma Subjects

Study Overview
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Brief Summary
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This is a single arm, open-label, uni-center, phase I/II study to determine the safety and efficacy of an experimental therapy called BinD19 cells in childhood patients with B-cell acute lymphoblastic leukemia or lymphoma, who are chemo-refractory, relapsed after allogeneic SCT, or are otherwise ineligible for allogeneic stem cell transplant.

Official Title
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A Clinical Study Evaluating the Safety and Efficacy of BinD19 Treatment in Relapse or Refractory Childhood Acute Lymphoblastic Leukemia and Lymphoma Subjects

Conditions
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Relapsed B-cell Acute Lymphoblastic Leukemia, Childhood, Refractory B-cell Acute Lymphoblastic Leukemia, Childhood, Relapsed/Refractory B-cell Lymphoma, Childhood

Intervention / Treatment
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* Biological: BinD19


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female subjects with CD19+ B cell malignancies in patients with no available curative treatment options (such as autologous or allogeneic SCT) who have limited prognosis (several months to < 2 year survival) with currently available therapies will be enrolled. CD19+ leukemia or lymphoma Not eligible for allogeneic SCT because of age, comorbid disease, or lack of available family member or unrelated donor Follicular lymphoma, previously identified as CD19+: Disease responding or stable after most recent therapy (chemotherapy, MoAb, etc) ECOG result is 0, 1 or 2. With normal heart, liver and kidney functions. Negative serum DNA for EBV and CMV; negative antigen for HBV; negative serum antibody for HCV, HIV and syphilis. Negative in pregnancy test (female subject only). Exclusion Criteria: ECOG result is 3, 4 or 5. Pregnant or lactating female Uncontrolled active infection Active hepatitis B or hepatitis C infection Concurrent use of systemic steroids. Recent or current use of inhaled steroids is not exclusionary Previously treatment with any gene therapy products HIV infection Enrolled to other clinical study in the last 4 weeks. Subjects with systemic auto-immune disease or immunodeficiency. Subjects with CNS diseases. Subjects with secondary tumors. Subjects with tumor infiltration in liver, brain or GI tract.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 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: BinD19<br>BinD19 (autologous T cells transduced with CD19 TCR-ζ/4-1BB vector) administered as an IV infusion on days 0, 1 and 2 in the absence of disease progression or unacceptable toxicity. Minimum/ maximum dose: 1x10^6/kg / 1x10^7/kg administered to childhood patients with R/R B cell Acute Lymphoblastic Leukemia (ALL) or Lymphoma. | Biological: BinD19<br>* Autologous T cells purified from the peripheral blood mononuclear cells of subjects, transduced with TCR- ζ/4-1BB lentiviral vector, expanded in vitro and then frozen for future administration.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events [Safety and Feasibility] | To evaluate the safety and feasibility of BinD19 CAR-T cells in patients with refractory /relapsed B-cell acute lymphoblastic leukemia or lymphoma. | Study treatment until Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Response [Efficacy] | Efficacy assessments for ALL were performed based on bone marrow and blood morphologic criteria and physical examination findings. Efficacy assessments for Lymphoma were based on tumor load, B cell number and immunoglobulins. | 5 years |