NCT06609928

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

Recruiting now Phase 1 Last updated 12 January 2026

What this trial tests

Phase 1 trial testing FOLR1 CAR T-cells in Recurrent Childhood Acute Myeloid Leukemia in 12 participants. Currently enrolling.

Timeline

24 February 2025

Primary endpoint
1 October 2027

1 October 2042

Quick facts

Lead sponsor	Fred Hutchinson Cancer Center
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	24 February 2025
Primary completion	1 October 2027
Estimated completion	1 October 2042
Sites	1 location across United States

Drugs / interventions tested

• FOLR1 CAR T-cells — full drug profile →
• Biospecimen Collection — full drug profile →
• Bone Marrow Aspiration — full drug profile →
• Bone Marrow Biopsy
• Cyclophosphamide (cyclophosphamide) — full drug profile →
• Echocardiography Test
• Fludarabine (FLUDARABINE) — full drug profile →
• Pheresis
• Positron Emission Tomography

Conditions studied

• Recurrent Childhood Acute Myeloid Leukemia — all drugs for Recurrent Childhood Acute Myeloid Leukemia →
• Refractory Childhood Acute Myeloid Leukemia — all drugs for Refractory Childhood Acute Myeloid Leukemia →

Sponsor

Fred Hutchinson Cancer Center — full company profile →

Who can join

Under 6, any sex, with Recurrent Childhood Acute Myeloid Leukemia or Refractory Childhood Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. TP53 -Mutated Myeloid Neoplasms: 2024 Update on Diagnosis, Risk-Stratification, and Management.
   Shah MV, Arber DA, Hiwase DK. · · 2025 · cited 15× · PMID 40066944 · DOI 10.1002/ajh.27655
2. CAR-T and CAR-NK cell therapies in AML: breaking barriers and charting the future.
   Wu H, Shafiei FS, Taghinejad Z, Maleknia M, et al · · 2025 · cited 4× · PMID 41131610 · DOI 10.1186/s12967-025-07151-5
3. Have CARs stalled for non-B-cell malignancies? Where are we, and where are we going?
   Silbert S, Lamble A. · · 2025 · PMID 41347986 · DOI 10.1182/hematology.2025000733
4. Preclinical Evaluation of Folate Receptor-α Chimeric Antigen Receptor T Cells Exhibits Highly Efficient Antitumor Activity against Osteosarcoma.
   Choe M, Kirkey D, Lira I, Lira I, et al · · 2025 · PMID 40919994 · DOI 10.1158/2767-9764.crc-25-0086