FH-FOLR1 CAR T-cell Therapy for Childhood AML
This study is testing a new treatment called FH-FOLR1 CAR T-cells for children up to 6 years old with acute myeloid leukemia (AML) that has returned or not responded to previous treatments. CAR T-cell therapy uses your own immune cells (T cells) that are specially modified in the lab to find and attack cancer cells that have a specific marker called FOLR1. The main goals are to see how safe this treatment is and if the FH-FOLR1 CAR T-cells can be successfully made. To join, your child must be 6 years old or younger, weigh at least 7 kilograms, and have AML that shows the FOLR1 marker. This is a dose-escalation study, meaning different doses of the treatment will be tested.
- Study design
- This is a dose-escalation study, meaning different doses of the treatment will be tested. It plans to enroll 12 participants.
- What's involved
- Participants will undergo apheresis to collect T cells, receive chemotherapy (fludarabine and cyclophosphamide), and then receive the FH-FOLR1 CAR T-cells intravenously. They will also have cerebrospinal fluid (CSF) and blood samples collected, bone marrow aspirations and biopsies, and may have imaging like PET scans.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 15 years after treatment.
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NCT06609928
FH-FOLR1 Chimeric Antigen Receptor T Cell Therapy for Treating Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
Recruiting
PHASE1Up to 6InterventionalTreatmentFred Hutchinson Cancer CenterInvestigator-initiated
~12 participants
Updated 2026-08-06 on ClinicalTrials.gov
What's tested:FOLR1 CAR T-cellsBiospecimen CollectionBone Marrow AspirationBone Marrow BiopsyCyclophosphamideEchocardiography Test
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of adverse events
Measured over Up to 15 years
+1 more outcome measured
Conditions
Where it's being run
1 sites across 1 statesWashington1
Study leadership
- Katherine G. Tarlock, MD · PRINCIPAL_INVESTIGATOR · Fred Hutch/University of Washington/Seattle Children's Cancer Consortium
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Subject age ≤ 6 years.
Weight ≥ 7 kilograms.
AML that expresses FOLR1 by flow cytometry as assessed by Hematologics, Inc.
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 cycle of therapy).
Second or greater relapse of AML.
Refractory AML, defined as ≥ 0.1% leukemic cells determined by flow cytometry or \> 1% on biopsy after 2 cycles of chemotherapy.
Able to tolerate apheresis.
Life expectancy ≥ 8 weeks.
Has an appropriate stem cell donor source identified.
Lansky performance status score of ≥ 50. Subjects who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for purposes of assessing performance status.
The subject must discontinue all anticancer agents and radiotherapy and, in the opinion of the investigator, have fully recovered from significant acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy:
Chemotherapy and biologic agents: All chemotherapy and biologic therapy not specifically mentioned below must be discontinued ≥ 14 days prior to enrollment, with the exception of intrathecal chemotherapy for which there is not a required washout period.
Steroid use: All corticosteroid therapy (unless physiologic replacement dosing) must be discontinued ≥ 7 days prior to enrollment, unless being used to treat graft-versus-host disease (GVHD) (if being used to treat GVHD see requirements).
Tyrosine kinase inhibitor (TKI) use: All TKIs must be discontinued ≥ 3 days prior to enrollment.
Hydroxyurea: must be discontinued ≥ 1 day prior to enrollment.
FOLR1 targeting therapy must be discontinued within 30 days prior to enrollment.
Gene modified cellular therapy:
Must be at least 30 days from most recent gene modified cell therapy infusion and document no evidence of modified cells in the peripheral blood OR
Must be at least 60 days from most recent gene modified cell therapy.
Serum creatinine ≤ 1.5 x the upper limit of normal (ULN) based on the following:
Age 1 to \< 2 years: maximum serum creatinine 0.6 mg/dL for male and 0.6 mg/dL for female.
Age 2 to \< 6 years: maximum serum creatinine 0.8 mg/dL for male and 0.8 mg/dL for female.
Age 6 to \< 10 years: maximum serum creatinine 1 mg/dL for male and 1 mg/dL for female.
Total bilirubin ≤ 3 times ULN for age OR conjugated bilirubin ≤ 2 mg/dL.
Alanine aminotransferase (ALT)(serum glutamic-pyruvic transaminase \[SGPT\]) ≤ 5 times ULN.
Shortening fraction ≥ 28% OR ejection fraction (EF) ≥ 50% as measured by echocardiogram.
Oxygen saturation ≥ 92% on room air without supplemental oxygen or mechanical ventilation.
Absolute lymphocyte count (ALC) ≥ 100 cells/uL.
Virology testing negative within 3 months prior to enrollment, to include:
HIV antigen \& antibody.
Hepatitis B surface antigen.
Hepatitis C antibody OR if positive, hepatitis C polymerase chain reaction (PCR) is negative.
Subject and/or legally authorized representative has signed the informed consent form for this study.
Exclusion
Active malignancy other than acute myeloid leukemia.
History of symptomatic non-AML central nervous system (CNS) disease or ongoing symptomatic CNS disease requiring medical intervention, including paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injury, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder (subjects with non-febrile seizure disorder controlled on anti-epileptic medication and without seizure activity within 1 month are eligible).
CNS AML involvement that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and T cell infusion.
If history of allogeneic stem cell transplant: active GVHD or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks prior to enrollment.
If history of allogeneic stem cell transplant and patient has received donor lymphocyte infusion (DLI) the subject is \< 8 weeks from DLI infusion.
Presence of active severe infection, defined as:
Positive blood culture within 48 hours of enrollment, OR
Fever above 38.2 degrees Celsius (C), AND clinical signs of infection within 48 hours of enrollment.
Primary immunodeficiency syndrome.
Subject has received prior virotherapy.
Subject and/or legally authorized representative unwilling to provide consent/assent for participation in the 15-year follow-up period, required if FH-FOLR1 CAR T cell therapy is administered.
Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol.
Considered by the investigator to be unable to tolerate a lymphodepleting regimen.
What this trial measures
- Incidence of adverse eventsUp to 15 years
Will be summarized in terms of type, severity, date of onset, and attribution using the Common Terminology for Adverse Events version 5.
- Rate of manufacturing anti-FOLR1 chimeric antigen receptor (CAR) T-cells (FH-FOLR1 CAR T) productUp to 28 days
Feasibility will be determined by the rate of manufacturing a FH-FOLR1 CAR T cell product from apheresis product.