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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT06865664
Phase
PHASE1
Status
Recruiting
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Conditions

  • Rhabdomyosarcoma

Eligibility Criteria

Sex
ALL
Age
3 Years - 39 Years
Healthy Volunteers
Not accepted

Interventions

  • fludarabine — DRUG
    30 mg/m2 per day IV on days -5, -4, -3, -2
  • cyclophosphamide — DRUG
    500 mg/m2 per day IV on days -4, -3, -2
  • cetuximab — DRUG
    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.
  • FGFR4-CAR T Cells — BIOLOGICAL
    Single intravenous (IV) infusion on Day 0

Study Details

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 Dates

Start date
Sep 22, 2025
Status verified
Mar 2026
Primary completion
Dec 1, 2027
Completion
Apr 1, 2029

Study Design

Enrollment
50 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Arm 1
    Lymphodepleting preparative regimen (fludarabine and cyclophosphamide) followed by infusion of FGFR4-CAR T cells escalation/de-escalation dose levels
  • Experimental: Arm 2
    Lymphodepleting preparative regimen (fludarabine and cyclophosphamide) followed by infusion of FGFR4-CAR T cells at the MTD

Primary Outcome Measure

Estimate the MTD of FGFR4-CAR T cells in children and young adults with recurrent or refractory rhabdomyosarcoma following a cyclophosphamide/fludarabine lymphodepletion regimen [ Time Frame: 28 days ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
Jo Hurtt, B.S.N.
[email protected]
240-858-7012
Srivandana Akshintala, M.D.
[email protected]
(240) 858-3448

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