Residual Disease Driven Strategy for CARCIK (CD19) in Adults/Pediatric BCP-ALL

Study Overview
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Brief Summary
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This is a single arm, open-label, multi-center, phase II study to determine the activity and the safety of a therapeutic strategy that allows a second CARCIK-CD19 cells infusion, driven by the status of disease from one month after the first infusion, in adult and pediatric patients with r/r BCP- ALL.

Official Title
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Measurable Residual Disease Driven Strategy for One or Two Infusions of Non- Viral, Transposon-manipulated CARCIK (CD19) Cells: A Phase II Study in Pediatric and Adult Patients With Relapsed/Refractory B Cell Precursor ALL (BCP-ALL)

Conditions
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Acute Lymphoblastic Leukemia

Intervention / Treatment
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* Genetic: PTG-CARCIK-CD19


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children (1-17) and adults (18-75 years old); Relapsed or refractory adult and pediatric B- ALL as defined for the presence of bone marrow with ≥ 5% lymphoblasts by morphologic assessment, or if <5%, with at least 1% of molecular disease at PCR; Evidence of CD19 tumor expression in bone marrow and/or peripheral blood by flow cytometry; Diagnosis of CD19 positive ALL in the bone marrow, and/or peripheral blood and/or extramedullary sites with the exclusion of Central Nervous System (CNS) if CNS-3 disease. Exclusion Criteria: GVHD Grades II-IV for patients who had previously been transplanted; 2. Any cell therapy in the previous 30 days; Patient with concomitant life-threatening infectious disease; Lansky/Karnofsky score <60; Patients with hepatic or renal disease as specific above; Pregnant or breast-feeding females; Rapidly progressive disease that in the estimation of the investigator and sponsor would compromise ability to complete study therapy;

Ages Eligible for Study
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Minimum Age: 1 Year
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: CARCIK-CD19<br> | Genetic: PTG-CARCIK-CD19<br>* PTG-CARCIK-CD19 cells is a gene therapy medicinal product (GTMP) composed of genetically modified allogeneic T lymphocytes formulated as cell suspension in normal saline solution and freezing media<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the overall response rate: change from baseline at day 28 after the first CARCIK-CD19 infusion | At baseline and at Day 28 after infusion: lymphoblasts < 5% at morphological evaluation of bone marrow aspirate | Baseline and day 28 | 
| To evaluate the overall response rate: change from baseline at day 28 after the first CARCIK-CD19 infusion | At baseline and at Day 28 after infusion: lymphoblasts < 5% at flow cytometry bone marrow evaluation | Baseline and day 28 | 
| To evaluate the overall response rate: change from baseline at day 28 after the first CARCIK-CD19 infusion | At baseline and at Day 28 after infusion: molecular evaluation of disease positivity <0.01% | Baseline and day 28 | 
| To evaluate the duration of response of patients treated with CARCIK - CD19 cells. Change from day 28 at month 2, 3, 4, 5, 6, 9 and 12 | Day 28 and month 2, 3, 4, 5, 6, 9 and 12: lymphoblasts < 5% at morphological evaluation of bone marrow aspirate | Day 28 and month 2, 3, 4, 5, 6, 9 and 12 | 
| To evaluate the duration of response of patients treated with CARCIK - CD19 cells. Change from day 28 at month 2, 3, 4, 5, 6, 9 and 12 | Day 28 and month 2, 3, 4, 5, 6, 9 and 12: lymphoblasts < 5% at flow cytometry bone marrow evaluation | Day 28 and month 2, 3, 4, 5, 6, 9 and 12 | 
| To evaluate the duration of response of patients treated with CARCIK - CD19 cells. Change from day 28 at month 2, 3, 4, 5, 6, 9 and 12 | Day 28 and month 2, 3, 4, 5, 6, 9 and 12: molecular evaluation of disease positivity <0.01% | Day 28 and month 2, 3, 4, 5, 6, 9 and 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALL