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Clinical trial researching CAR T-cell therapy for rhabdomyosarcoma

Doctor with young patient.

Doctor with young patient. Image credit: Canva

Rhabdomyosarcoma (RMS) is a cancer of soft tissues; it is the most common soft tissue sarcoma seen in children. Researchers want to try approaching rhabdomyosarcoma with chimeric antigen receptor (CAR) T-cell therapy, in which a person’s own T cells are collected, and then modified so they are better able to target a protein, FGFR4, on the surface of RMS tumor cells.

A clinical trial led by Srivandana Akshintala, M.B.B.S., M.P.H., Assistant Research Physician in the Pediatric Oncology Branch, is researching FGFR4-CAR T-cell therapy for children and young adults with rhabdomyosarcoma.

The trial will take place at the NIH Clinical Center in Bethesda, Maryland, and there is no cost for medical care received at the Clinical Center.

For more information, please contact the NCI Cancer Information Service at 1-888-624-1937 or nciinfo@mail.nih.gov

Clinicaltrials.gov identifier: NCT06865664

NCI Protocol ID: IRB001570

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

The Center for Cancer Research is NCI’s internal cancer center, a publicly funded organization working to improve the lives of cancer patients by solving important, challenging and neglected problems in cancer research and patient care. Highly trained physician-scientists develop and carry out clinical trials to create the medicines of tomorrow treating patients at the world’s largest dedicated research hospital on the campus of the National Institutes of Health in Bethesda, Maryland.

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Posted on Fri, 08/08/2025