FH-FOLR1 Chimeric Antigen Receptor T Cell Therapy for Treating Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT ID: NCT06609928
Last Updated: 2026-01-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1
12 participants
INTERVENTIONAL
2025-02-24
2042-10-01
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Fludarabine Phosphate, Cyclophosphamide, Total-Body Irradiation, and Donor Bone Marrow Transplant Followed by Donor Natural Killer Cell Therapy, Mycophenolate Mofetil, and Tacrolimus in Treating Patients With Hematologic Cancer
NCT00789776
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
NCT02566304
Fludarabine Phosphate, Low-Dose Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies or Kidney Cancer
NCT00005799
Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine, Mycophenolate Mofetil, Donor Lymphocyte Infusion in Treating Patients With Hematopoietic Cancer
NCT00006251
Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia
NCT00860574
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
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
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Treatment (FH-FOLR1 CAR T)
Patients 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.
FOLR1 CAR T-cells
Given IV
Biospecimen Collection
Undergo CSF and blood sample collection
Bone Marrow Aspiration
Undergo bone marrow aspiration
Bone Marrow Biopsy
Undergo bone marrow biopsy
Cyclophosphamide
Given IV
Echocardiography Test
Undergo ECHO
Fludarabine
Given IV
Pheresis
Undergo apheresis
Positron Emission Tomography
Undergo PET
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
FOLR1 CAR T-cells
Given IV
Biospecimen Collection
Undergo CSF and blood sample collection
Bone Marrow Aspiration
Undergo bone marrow aspiration
Bone Marrow Biopsy
Undergo bone marrow biopsy
Cyclophosphamide
Given IV
Echocardiography Test
Undergo ECHO
Fludarabine
Given IV
Pheresis
Undergo apheresis
Positron Emission Tomography
Undergo PET
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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 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 Criteria
* 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.
6 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Kuni Foundation
UNKNOWN
Fred Hutchinson Cancer Center
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Katherine G. Tarlock, MD
Role: PRINCIPAL_INVESTIGATOR
Fred Hutch/University of Washington/Seattle Children's Cancer Consortium
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Fred Hutch/University of Washington/Seattle Children's Cancer Consortium
Seattle, Washington, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
NCI-2024-06808
Identifier Type: REGISTRY
Identifier Source: secondary_id
STUDY00004888
Identifier Type: OTHER
Identifier Source: secondary_id
RG1124385
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.