FGFR4 CAR T-cell Therapy for Rhabdomyosarcoma
This study is testing a new type of treatment called FGFR4-CAR T cells for children and young adults (ages 3-39) with rhabdomyosarcoma (a soft tissue cancer) that has returned or not responded to at least two previous treatments. Rhabdomyosarcoma cells have a protein called FGFR4 on their surface. Researchers will collect your own immune cells (T cells), modify them in the lab to specifically target FGFR4, and then give them back to you. Before receiving the FGFR4-CAR T cells, you will get chemotherapy with fludarabine and cyclophosphamide, and some participants will also receive cetuximab. The main goal is to find the safest and most effective dose of FGFR4-CAR T cells.
- Study design
- This is an interventional study with a planned enrollment of 50 participants. The phase is not specified, and the current status is unclear.
- What's involved
- Participants will receive chemotherapy (fludarabine and cyclophosphamide) for several days, followed by a single infusion of FGFR4-CAR T cells. Some participants will also receive weekly infusions of cetuximab for four weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- The main goal of the study, finding the best dose of FGFR4-CAR T cells, will be measured at 28 days after treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT06865664
FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma
Recruiting
PHASE1Ages 3–39InterventionalTreatmentNational Cancer Institute (NCI)Government-funded
~50 participants
Updated 2026-07-21 on ClinicalTrials.gov
What's tested:fludarabinecyclophosphamidecetuximabFGFR4-CAR T Cells
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Estimate the MTD of FGFR4-CAR T cells in children and young adults with recurrent or refractory rhabdomyosarcoma following a cyclophosphamide/fludarabine lymphodepletion regimen
Measured over 28 days
Conditions
Where it's being run
1 sites across 1 statesMaryland1
Study leadership
- Srivandana Akshintala, M.D. · PRINCIPAL_INVESTIGATOR · National Cancer Institute (NCI)
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Histologically confirmed rhabdomyosarcoma by the NCI Department of Pathology.
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).
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
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)
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
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
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
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
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.
What this trial measures
- Estimate the MTD of FGFR4-CAR T cells in children and young adults with recurrent or refractory rhabdomyosarcoma following a cyclophosphamide/fludarabine lymphodepletion regimen28 days
Estimation of MTD using evaluation of adverse events considered to be a DLT within DLT period as explained in sections Dose Limiting Toxicity (DLT) and Dose Escalation (Arm 1)