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Engineered immune cells take on childhood cancer

NCT ID NCT06865664

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 15, 2026 · Updated 4 times

Summary

This early-phase trial tests a new treatment for children and young adults with rhabdomyosarcoma that has come back or not responded to standard therapy. The treatment uses the patient's own immune cells, which are modified in a lab to better target cancer cells. Up to 50 participants will receive the modified cells along with chemotherapy to prepare their bodies, and researchers will monitor safety and the right dose.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
FGFR4-CAR T cells (a type of modified immune cell)
What this could lead to
If it works, this could point toward a new treatment option for children with hard-to-treat rhabdomyosarcoma.
What could go wrong
This is a very early Phase 1 trial with only 50 participants, so it may not work or could have serious side effects. The modified cells might not control the cancer long-term.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2025

Expected to finish

Apr 2029

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 39 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Email: •••••@•••••

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