Actively Recruiting
FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma
Led by National Cancer Institute (NCI) · Updated on 2026-03-16
50
Participants Needed
1
Research Sites
183 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed rhabdomyosarcoma
- Relapsed or refractory rhabdomyosarcoma after at least two cancer treatment regimens
- No alternative curative therapies available
- Measurable or non-measurable disease on imaging
- Age between 3 and 39 years
- Weight at least 15 kg
- Performance status: Karnofsky or Lansky score 50% or higher
- Willingness to accept blood transfusions
- Adequate organ and marrow function including specific blood counts, liver, kidney, heart, lung, and neurological criteria
- Agreement to use effective contraception if of child-bearing potential
- Willingness to discontinue nursing during treatment
- Stable previous CNS tumor involvement if applicable
- Willingness to enroll in long-term follow-up study after 5 years
- Ability to understand and sign informed consent
You will not qualify if you...
- Recent therapy within defined time frames before apheresis including tyrosine kinase inhibitors, chemotherapy, antibodies, radiation, investigational agents, stem cell transplant, or genetically modified cell therapies
- Use of systemic steroids above physiological doses
- History of severe allergic reactions to study agents or cell manufacturing components
- Second malignancy
- Primary immunodeficiency
- Positive for HIV, hepatitis B or C infections
- Pregnancy confirmed by testing
- Uncontrolled illness or social situations limiting study compliance
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
N
NCI POB Solid Tumor Referral Team
CONTACT
S
Srivandana Akshintala, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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