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New treatment option for Recurrent Childhood Acute Myeloid Leukemia
Official title FH-FOLR1 Chimeric Antigen Receptor T Cell Therapy for Treating Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
ClinicalTrials.gov ID: NCT06609928
What this study is testing
What is FOLR1 CAR T-cells?
FOLR1 CAR T-cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for recurrent childhood acute myeloid leukemia.
Also referred to as Anti-FOLR1 CAR-T Cells, FH-FOLR1 CAR T Cells.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 6
You may be able to join if
- 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 people who have previously received an allogeneic hematopoietic cell transplantation (HCT), any evidence of AML re-emergence post HCT detectable...
- First relapse of AML ≤ 6 months from initial diagnosis.
You likely can't join if
- 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...
- CNS AML involvement that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and T...
- If history of allogeneic stem cell transplant: active GVHD or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks...
- 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:
See the full eligibility criteria
Who can 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 people 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. people 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...
- 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...
- 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...
- 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.
What rules you out
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Seattle, Washington, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 6 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Seattle, Washington, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.