This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19 in pediatric and young adult patientswith hypodiploid (Cohort A) or t(17;19) B-ALL (Cohort B), infants with very high risk KMT2A B-ALL (Cohort C), and in patients with central nervous system (CNS) relapse who did not receive cranial radiation (XRT) or bone marrow transplantation (BMT) (Cohort D).
Signed informed consent form must be obtained prior to any study procedure.
Male and female patients with documented CD19+ B-ALL
Documentation of CD19 tumor expression in bone marrow, peripheral blood, CSF, or tumor tissue.
Age 0 to 29 years
For subjects with a CNS relapse, prior cranial XRT or BMT for the current relapse is an exclusion.
Active hepatitis B or active hepatitis C.
HIV Infection.
Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
Parallel
Murine CART19
Biological/VaccineCART19 cells transduced with a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ, administered by IV injection with a planned dose of 5x106 CART19 cells/kg on day 0 with possible reinfusion/retreatment
Event-free survival (EFS)
1 year event-free survival (EFS), where events include no response, relapse, death due to any cause
EFS Rate 1
Modified EFS rate in CNS relapse patients, using a definition of events that includes no response, relapse, death, need for XRT or need for BMT
To further evaluate the safety of CART19 in the target patient populations
Frequency and severity of adverse events
EFS rate 2
Modified EFS rate in patients with early CNS relapsed B-ALL (CR1 \<18 months) and those with late CNS relapsed B-ALL (CR1 \>18 months) using a definition of events that includes no response, relapse, death, need for XRT or need for BMT
MRD conversion
Rate of MRD conversion to less than 0.01% (in patients with MRD) 28 days after CART19 therapy in patients with t(17;19) B-ALL, hypodiploid B-ALL, and very high risk infant B-ALL
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Stephan Grupp MD PhD
Lead sponsor
Children's Hospital of Philadelphia
Sponsor institution
University of Pennsylvania
Collaborator
