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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 1INTERVENTIONAL

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

FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma (NCT06865664) is a Phase 1 interventional studying Rhabdomyosarcoma, sponsored by National Cancer Institute (NCI). RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Rhabdomyosarcoma, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 50 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Rhabdomyosarcoma subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

* INCLUSION CRITERIA - diagnosed by tissue sample (biopsy-confirmed) rhabdomyosarcoma by the NCI Department of Pathology. Note: Since FGFR4 expression is universal in rhabdomyosarcoma, confirmation of FGFR4 expression is not required. - Relapsed or refractory rhabdomyosarcoma after at least two (2) cancer treatment regimens i.e., participants should have relapsed or progressed after upfront therapy (that includes any systemic chemotherapy with or without local control) as well as at least one salvage therapy (which can be systemic therapy, radiation, or surgery). - No available alternative curative therapies per standard of care. - Participants must have tumors that can be measured on scans 1.1 or non-measurable disease on imaging. - Age \>= 3 and \<= 39 years old. - Weight \>=15 kg. - Performance status: Karnofsky \>= 50% (\>= 16 years) or Lansky \>= 50% (\< 16 years). Note: Participants who are unable to walk because of paralysis, but who are upright in a wheelchair will be considered ambulatory for calculating the performance score. - Participants must be willing to accept blood transfusions. - Adequate organ and marrow function as defined below: - Organ: Bone Marrow Function\* - Laboratory Element: Absolute neutrophil count; Minimum Requirement \>= 500/mcL - Laboratory Element: Platelets; Minimum Requirement \>= 50,000/mcL \*Transfusion independent (defined as no transfusion in the prior 7 days) for participants without bone marrow involvement. Participants who have bone marrow involvement with tumor are exempt from the platelet requirement and will not be evaluable for hematological toxicities. Participants must not be refractory to transfusions. - Organ: Liver Function - Laboratory Element: Aspartate aminotransferase (AST); Minimum Requirement \<= 5 x upper limit of normal (ULN) - Laboratory Element: Alanine aminotransferase (ALT); Minimum Requirement \<= 5 x ULN ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
* INCLUSION CRITERIA * Histologically confirmed rhabdomyosarcoma by the NCI Department of Pathology. Note: Since FGFR4 expression is universal in rhabdomyosarcoma, confirmation of FGFR4 expression is not required. * Relapsed or refractory rhabdomyosarcoma after at least two (2) cancer treatment regimens i.e., participants should have relapsed or progressed after upfront therapy (that includes any systemic chemotherapy with or without local control) as well as at least one salvage therapy (which can be systemic therapy, radiation, or surgery). * No available alternative curative therapies per standard of care. * Participants must have measurable disease per RECIST 1.1 or non-measurable disease on imaging. * Age \>= 3 and \<= 39 years old. * Weight \>=15 kg. * Performance status: Karnofsky \>= 50% (\>= 16 years) or Lansky \>= 50% (\< 16 years). Note: Participants who are unable to walk because of paralysis, but who are upright in a wheelchair will be considered ambulatory for calculating the performance score. * Participants must be willing to accept blood transfusions. * Adequate organ and marrow function as defined below: * Organ: Bone Marrow Function\* * Laboratory Element: Absolute neutrophil count; Minimum Requirement \>= 500/mcL * Laboratory Element: Platelets; Minimum Requirement \>= 50,000/mcL \*Transfusion independent (defined as no transfusion in the prior 7 days) for participants without bone marrow involvement. Participants who have bone marrow involvement with tumor are exempt from the platelet requirement and will not be evaluable for hematological toxicities. Participants must not be refractory to transfusions. * Organ: Liver Function * Laboratory Element: Aspartate aminotransferase (AST); Minimum Requirement \<= 5 x upper limit of normal (ULN) * Laboratory Element: Alanine aminotransferase (ALT); Minimum Requirement \<= 5 x ULN * Laboratory Element: Total bilirubin; Minimum Requirement \<= 2 x ULN (Note: Participants with Gilbert's syndrome and/or bilirubin elevation due to tumor involvement are allowed to have \<= 5 x ULN) Note: Adult values will be used for calculating hepatic toxicity and determining eligibility --Organ: Renal Function * Age: 3 to \< 6 years; Maximum serum creatinine (mg/dL): Male - 0.8, Female - 0.8 * Age: 6 to \< 10 years; Maximum serum creatinine (mg/dL): Male - 1, Female - 1 * Age: 10 to \< 13 years; Maximum serum creatinine (mg/dL): Male - 1.2, Female - 1.2 * Age: 13 to \< 16 years; Maximum serum creatinine (mg/dL): Male - 1.5, Female - 1.2 * Age: \>= 16 years; Maximum serum creatinine (mg/dL): Male - 1.7, Female - 1.4 OR * Measured or calculated creatinine clearance or glomerular filtration rate (GFR); Minimum Requirement: \>= 60mL/min/1.73 m\^2 --Organ: Cardiac Function * Laboratory Element: Cardiac status; Minimum Requirement: Cardiac ejection fraction \>= 45% or shortening fraction \>= 28%, pericardial effusion \<= grade 2 as determined by an echocardiogram (ECHO) * Organ: Pulmonary Function * Laboratory Element: Pulmonary status; Minimum Requirement: Pleural effusion \<= grade 1; Oxygen (O2) saturation \>=92% on room air at rest --Organ: Neurological Function * Laboratory Element: Neurologic status; Minimum Requirement: No acute neurotoxicity greater than grade 2 per CTCAE v.5.0 with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible. * Individuals of child-bearing potential (IOCBP) must agree to use highly effective contraception (hormonal, intrauterine device \[IUD\], abstinence, surgical sterilization) at the study entry and up to 12 months after the last dose of combined chemotherapy or 6 months after FGFR4-CAR T cells infusion, whichever is later. Individuals who can father children must agree to use an effective method of contraception (barrier, surgical sterilization, abstinence) at the study entry and up to 4 months after the last dose of combined chemotherapy or 6 months after FGFR4-CAR T cells infusion, whichever comes later. We also will recommend individuals who can father children with IOBCP partners ask their partners to be on highly effective birth control (hormonal, IUD, surgical sterilization). Individuals who can father children must not freeze or donate sperm within the same period. * Nursing participants must be willing to discontinue nursing from study treatment initiation through 4 months after completion of chemotherapy preparative administration or 6 months after FGFR4-CAR T cells infusion, whichever is later. * Participants with previous central nervous system (CNS) tumor involvement that has been treated and is stable for at least 6 weeks following completion of therapy as evidenced by no requirements for corticosteroids, no evolving neurologic deficits, and no progression of residual brain abnormalities without specific therapy, are permitted. Participants with asymptomatic subcentemeric CNS lesions are permitted if no immediate radiation or surgery is indicated. * Participants must be willing to be enrolled into protocol 15C0028 "Follow-Up Evaluation for Gene-Therapy Related Delayed Adverse Events after Participation in Pediatric Oncology Branch Clinical Trials" after 5 years on this trial. * The ability of participant or parent/guardian to understand and the willingness to sign a written informed consent document. EXCLUSION CRITERIA * 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.

Treatments Being Tested

DRUG

fludarabine

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

DRUG

cyclophosphamide

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

DRUG

cetuximab

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.

BIOLOGICAL

FGFR4-CAR T Cells

Single intravenous (IV) infusion on Day 0

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

National Institutes of Health Clinical Center
Bethesda, Maryland, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06865664), the sponsor (National Cancer Institute (NCI)), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06865664 clinical trial studying?

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 an… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06865664?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06865664?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06865664. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06865664. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.