FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma
Recruiting now · Phase 1
Conditions studied: Rhabdomyosarcoma
In brief
Background: Rhabdomyosarcoma (RMS) is a cancer of soft tissues. It is the most common soft tissue sarcoma seen in children. RMS cancer cells have a protein called FGFR4 on their surface. Researchers want to try a new kind of treatment for RMS: They will collect a person s own T cells, a type of immune cell; then they will change the T cells so they are better able to target the FGFR4 protein and attack RMS tumor cells. The modified T cells are chimeric antigen receptor (CAR) T cells. The treatment in this study is called FGFR4-CAR T cells. Objective: To test FGFR4-CAR T cells in children and young adults with RMS. Eligibility: People aged 3 to 39 years with RMS. The RMS must have failed to respond or returned after at least 2 rounds of standard treatment. Design: Participants will be screened. They will have physical exam, imaging scans, blood tests, and tests of their heart. They may have a tissue sample taken from their tumor. They will undergo apheresis: Blood will be taken from the body through a catheter. The blood will pass through a machine that separates out the T cells, and the remaining blood will be returned to the body. The collected T cells will be taken to a lab to create FGFR4-CAR T cells. Once the FGFR4-CART cells are ready, participants can receive these T cells. For 4 days they will receive drugs to prepare their body for the FGFR4-CAR T cells. After this, the modified T cells will be infused into a vein. Participants will be then monitored closely to watch for any side effects from the CART cells and be followed to see what effect the CART cells have on their tumors. They will have follow-up visits for up to 5 years. Long-term follow-up will be another 10 years.
Key facts
- Study ID
- NCT06865664
- Run by
- National Cancer Institute (NCI)
- People needed
- 50
- Starts
- 2025-09-22
- Expected to finish
- 2029-04-01
- Last updated by the study team
- 2026-07-21
Who can join
Age: 3 and older, up to 39. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Prior therapy with the following prior to apheresis:
- tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen within <= 1 week
- systemic chemotherapy within <= 2 weeks
- antineoplastic antibody therapy, checkpoint inhibitors, or vaccine therapy, within <= 3 weeks or 5 half-lives (whichever is shorter)
- radiation within <= 3 weeks (<= 6 weeks if CNS or lung fields have been radiated or in case of craniospinal irradiation of radiation of >=50% of bony pelvis and <=12 weeks in case of total body irradiation). Note: There is no time restriction if the volume of bone marrow treated is less than 10% and the participant has measurable/evaluable disease outside the radiation port
- any investigational agents within <= 4 weeks
- autologous stem cell infusion following myeloablative therapy within <= 6 weeks
- genetically modified T cell, NK cell, or dendritic cell therapy within <= 6 weeks
- allogeneic stem cell transplant/infusion within <=12 weeks or evidence of active graft versus host disease (GVHD)
- Participants receiving more than physiologic dosing of systemic steroids (3 mg/m\^2/day of prednisone equivalent).
- History of severe, immediate hypersensitivity reaction attributed to any agents used in the study or in the manufacturing of the cells.
- Second malignancy at any time.
- Primary immunodeficiency.
- Seropositive for human immunodeficiency virus (HIV) antibody.
- Seropositive for hepatitis C (HCV) or positive for Hepatitis B (HBV) surface antigen (HbsAg).
- Pregnancy confirmed with beta-HCG serum or urine pregnancy test performed in IOCBP at screening.
- Uncontrolled intercurrent illness or social situations that would limit compliance with study requirements.
Where it is running
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)
Full record on ClinicalTrials.gov
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