NCT06609928

FH-FOLR1 Chimeric Antigen Receptor T Cell Therapy for Treating Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1 Study of FOLR1 CAR T for Pediatric Patients With FOLR1/CBFA2T3::GLIS2+ Relapsed or Refractory AML

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Detailed 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 are followed up for 15 years.

Conditions

• Recurrent Childhood Acute Myeloid Leukemia
• Refractory Childhood Acute Myeloid Leukemia

Interventions

Type	Name	Description
BIOLOGICAL	FOLR1 CAR T-cells	Given IV
PROCEDURE	Biospecimen Collection	Undergo CSF and blood sample collection
PROCEDURE	Bone Marrow Aspiration	Undergo bone marrow aspiration
PROCEDURE	Bone Marrow Biopsy	Undergo bone marrow biopsy
DRUG	Cyclophosphamide	Given IV
PROCEDURE	Echocardiography Test	Undergo ECHO
DRUG	Fludarabine	Given IV
PROCEDURE	Pheresis	Undergo apheresis
PROCEDURE	Positron Emission Tomography	Undergo PET

Timeline

• Start date: 2025-02-24
• Primary completion: 2027-10-01
• Completion: 2042-10-01
• First posted: 2024-09-24
• Last updated: 2026-01-12

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study