Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL
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This is a phase 2 study to evaluate humanized CD19 redirected autologous T cells (or huCART19 cells) with CD19 expressing relapsed and refractory B-cell acute lymphoblastic leukemia. This study is targeting pediatric and young adult patients aged 1-29 years with CD19+ B cell malignancies in newly diagnosed B-ALL patients predicted to have an exceedingly poor outcome with conventional chemotherapy, in high-risk first relapse, or and in second or greater relapse in this phase 2 trial. In addition, a second cohort will test the efficacy of huCART19 in patients with poor response to prior B cell directed engineered cell therapy.
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Layout table for eligibility information
Ages Eligible for Study:
1 Year to 29 Years (Child, Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Relapsed or refractory B-cell ALL:
Cohort A: Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL who meet one of the following criteria:
Newly diagnosed NCI HR B-ALL with induction failure: M3 marrow (>25% blasts) at end of induction OR
First marrow relapse of B-ALL at < 36 months from diagnosis OR
2nd or greater relapse OR
Any relapse after allogeneic hematopoietic stem cell transplantation (HSCT) and ≥ 4 months from stem cell transplant (SCT) at enrollment OR
Refractory disease defined as having not achieved a minimal residual disease (MRD)_-negative and/or cerebral spinal fluid (CSF)-negative complete response (CR) after ≥ 2 chemotherapy regimens/cycles of frontline therapy or 1 cycle of reinduction therapy for patients in first relapse OR
Ineligible for allogeneic stem cell transplant
Cohort B: Patients previously treated with B cell directed engineered cell therapy who meet one of the following criteria:
partial response or no response to prior cell therapy
CD19+ relapse after prior cell therapy
demonstrated early (≤6 months from infusion) B cell recovery suggesting loss of engineered cells
Patients with prior or current history of CNS3 disease will be eligible if central nervous system (CNS) disease is responsive to therapy
Documentation of CD19 tumor expression in bone marrow, peripheral blood, CSF, or tumor tissue by flow cytometry at relapse
Adequate organ function
Age 1-29 years
Adequate performance status
Active hepatitis B or active hepatitis C.
Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
Concurrent use of systemic steroids at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.
CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.