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Recurrent Childhood Acute Myeloid LeukemiaPhase 1NCT06609928

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

A Phase 1 study testing FOLR1 CAR T-cells, Biospecimen Collection and Bone Marrow Aspiration in people with Recurrent Childhood Acute Myeloid Leukemia and Refractory Childhood Acute Myeloid Leukemia, run by Fred Hutchinson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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). Read more about this study

Who is it for?
Ages Up to 6 years
What is being tested?
FOLR1 CAR T-cells, Biospecimen Collection and Bone Marrow Aspiration
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteUnited States
Study dates
24 Feb 2025to 1 Oct 2042
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 6 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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:
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Katherine G. Tarlock, MD contact

206-667-7121

katherine.tarlock@seattlechildrens.org

United States1 site, 1 recruiting

  • Fred Hutch/University of Washington/Seattle Children's Cancer Consortium

    Seattle, Washington, 98109

    Katherine G. Tarlock, MD, 206-667-7121, katherine.tarlock@seattlechildrens.org

    Katherine G. Tarlock, MD

    Recruiting

Other trials in United States

About this study

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

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.

Read the 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.

Being tested
FOLR1 CAR T-cells, Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Cyclophosphamide

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FOLR1 CAR T-cellsBiological

    Also called Anti-FOLR1 CAR-T Cells, FH-FOLR1 CAR T Cells

    Given IV

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo CSF and blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518

    Given IV

  • Echocardiography TestProcedure or surgery

    Also called EC, Echocardiography

    Undergo ECHO

  • FludarabineDrug

    Also called Fluradosa

    Given IV

  • PheresisProcedure or surgery

    Also called Apheresed, Apheresis, Blood Component Removal, Collection, Apheresis/Leukapheresis

    Undergo apheresis

  • Positron Emission TomographyProcedure or surgery

    Also called Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure)

    Undergo PET

Study groups in NCT06609928
Study groupRoleWhat they receive
Treatment (FH-FOLR1 CAR T)ExperimentalPatients undergo apheresis to obtain T cells for product manufacturing. Patients receive lymphodepleting chemotherapy with fludarabine IV on days -4 to -1 and cyclophosphamide IV on days -4 and -3. Patients receive FH-FOLR1 CAR T IV on day 0. Patients undergo ECHO at screening, undergo collection of CSF and blood samples and bone marrow aspiration/biopsy throughout the study, and may undergo PET scan on study and during follow up.
Receives: Biological: FOLR1 CAR T-cells; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Cyclophosphamide; Procedure: Echocardiography Test; Drug: Fludarabine; Procedure: Pheresis; Procedure: Positron Emission Tomography

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events

    Will be summarized in terms of type, severity, date of onset, and attribution using the Common Terminology for Adverse Events version 5.

    Measured: Up to 15 years

  2. Rate of manufacturing anti-FOLR1 chimeric antigen receptor (CAR) T-cells (FH-FOLR1 CAR T) product

    Feasibility will be determined by the rate of manufacturing a FH-FOLR1 CAR T cell product from apheresis product.

    Measured: Up to 28 days

Secondary outcomes (7)

  1. Aplasia

    Will be defined as if a patient has a hypoplastic bone marrow and has failure to recover a peripheral absolute neutrophil count > 200/uL and a non-transfusion dependent platelet count > 20,000/uL not due to malignant infiltration or severe infection (defined as ≥ grade 3 infection). Will be assessed using peripheral blood and bone marrow.

    Measured: At 42 days

  2. Persistence of FH-FOLR1 CAR T

    FOLR1 persistence will be defined as detection of the FH-FOLR1 CAR T by flow or polymerase chain reaction above the lower limit of detection. Will be assessed by peripheral blood.

    Measured: Up to 15 years

  3. Overall survival

    Measured: From infusion of FH-FOLR1 CAR T cell product to death from any cause, assessed up to 15 years

  4. Disease free survival

    Measured: From T cell infusion to the first observation of disease or death fromany cause, whichever occurs first, assessed up to 15 years

  5. Duration of overall response

    Measured: From the time criteria are met for complete response or partial response until the first date that treatment failure is objectively documented, assessed up to 15 years

  6. Non-relapse mortality

    Measured: From T cell infusion to death where cause of death is not attributable tounderlying disease, assessed up to 15 years

  7. Event free survival

    Measured: From infusion of FH-FOLR1 CAR T product to an event, with eventsdefined as relapse, secondary malignancy, death from any cause, assessed up to 15 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Sep 2024
  2. Study start24 Feb 2025
  3. Primary completion1 Oct 2027 (estimated)
  4. Study completion1 Oct 2042 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06609928
Sponsor's protocol ID
RG1124385
Other IDs
NCI-2024-06808, STUDY00004888
Status last verified
Aug 2026
First submitted
20 Sep 2024
Last updated
6 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Alfred JC, Geyer JK, Rodriguez A, Roata C, Buddenbaum J, Thompson PA, Wang JR, Alexander TB. Rapid Molecular Diagnostics Enables Individualized Frontline Treatment of CBFA2T3::GLIS2-Rearranged Acute Myeloid Leukemia. Pediatr Blood Cancer. 2026 Jul 25:e70584. doi: 10.1002/1545-5017.70584. Online ahead of print.

    PubMed 42500906

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing FOLR1 CAR T-cells, Biospecimen Collection and Bone Marrow Aspiration in people with Recurrent Childhood Acute Myeloid Leukemia and Refractory Childhood Acute Myeloid Leukemia, run by Fred Hutchinson Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 36 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Katherine G. Tarlock, MD (206-667-7121, katherine.tarlock@seattlechildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (time frame: Up to 15 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06609928, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2026.

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