RecruitingPhase 1NCT06865664

FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma

Phase I Dose Escalation Study of FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma


Sponsor

National Cancer Institute (NCI)

Enrollment

50 participants

Start Date

Sep 22, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Min Age: 3 YearsMax Age: 39 Years

Plain Language Summary

Simplified for easier understanding

This study tests a new type of cell therapy called CAR T cells — immune cells engineered to target and kill cancer — for children and young adults with rhabdomyosarcoma (a type of muscle cancer) that has come back or stopped responding to treatment. **You may be eligible if...** - You have been confirmed to have rhabdomyosarcoma by a pathologist - Your cancer has returned or stopped responding to treatment after at least two previous treatment regimens - You are 35 years old or younger - You are in adequate health (good organ function, no active serious infections) **You may NOT be eligible if...** - You have active brain or spinal cord involvement by cancer - You are pregnant or breastfeeding - You have had a bone marrow or stem cell transplant within the past 3 months - You have serious heart, lung, kidney, or liver problems Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGfludarabine

30 mg/m2 per day IV on days -5, -4, -3, -2

DRUGcyclophosphamide

500 mg/m2 per day IV on days -4, -3, -2

DRUGcetuximab

Participants Age \>=18 years, based on FDA approved dosing: Loading dose of 400 mg/m2 IV, followed by 250 mg/m2 IV weekly for a total of 4 doses. Participants Age \<18 years, based on phase I data of cetuximab in children: Dose of 250 mg/m2 IV administered over 1 hour weekly for a total of 4 doses.

BIOLOGICALFGFR4-CAR T Cells

Single intravenous (IV) infusion on Day 0


Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT06865664


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