A Phase 1 Study of FOLR1 CAR T for Pediatric Patients With FOLR1/CBFA2T3::GLIS2+ Relapsed or Refractory AML
Fred Hutchinson Cancer Center
Summary
This phase I trial tests the safety, side effects, and best dose of FH-FOLR1 chimeric antigen receptor (CAR) T cells in treating pediatric patients with FOLR1+ acute myeloid leukemia (AML) that has come back after a period of improvement (recurrent) or has not responded to previous treatment (refractory). CAR T-cell therapy is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a FOLR1 on the patient's cancer cells is added to the T cells in the laboratory. The special receptor is called a chimeric antigen receptor. Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. Chemotherapy drugs, such as fludarabine and cyclophosphamide, are given to a patient before the manufactured FH-FOLR1 CAR T cells are infused back into the patient to assist in the CAR T cell activity in the patient. The trial is evaluating if giving FH-FOLR1 CAR T cell therapy is safe and tolerable for pediatric patients with recurrent or refractory AML.
Description
OUTLINE: This is a dose-escalation study of FH-FOLR1 CAR T. Patients undergo apheresis to obtain T cells for product manufacturing, receive lymphodepleting chemotherapy with fludarabine intravenously (IV) on days -4 to -1, cyclophosphamide IV on days -4 and -3 and receive FH-FOLR1 CAR T IV on day 0. Patients undergo echocardiography (ECHO) at screening, undergo collection of cerebrospinal fluid (CSF), blood samples and bone marrow aspiration/biopsy throughout the study, and may undergo imaging (such as positron emission tomography (PET) scan). After completion of study treatment, patients ar…
Eligibility
- Age range
- Up to 6 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Subject age ≤ 6 years. * Weight ≥ 7 kilograms. * AML that expresses FOLR1 by flow cytometry as assessed by Hematologics, Inc. Laboratory and meets one of the below definitions: * For subjects who have previously received an allogeneic hematopoietic cell transplantation (HCT), any evidence of AML re-emergence post HCT detectable by flow cytometry. * First relapse of AML ≤ 6 months from initial diagnosis. * First relapse of AML \> 6 months from initial diagnosis with minimal residual disease (MRD) ≥ 0.05% by flow cytometry after at least one re-induction attempt (one cyc…
Interventions
- BiologicalFOLR1 CAR T-cells
Given IV
- ProcedureBiospecimen Collection
Undergo CSF and blood sample collection
- ProcedureBone Marrow Aspiration
Undergo bone marrow aspiration
- ProcedureBone Marrow Biopsy
Undergo bone marrow biopsy
- DrugCyclophosphamide
Given IV
- ProcedureEchocardiography Test
Undergo ECHO
- DrugFludarabine
Given IV
Location
- Fred Hutch/University of Washington/Seattle Children's Cancer ConsortiumSeattle, Washington