New immune cell therapy takes on childhood cancers that Won't quit
NCT ID NCT07172958
First seen Jun 24, 2026 · Last updated Aug 14, 2026 · Updated 3 times
Summary
This early-stage trial tests a new type of cell therapy for children whose embryonal tumors (rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor) have returned or not responded to standard treatments. The therapy uses the patient's own immune cells, engineered to better recognize and attack cancer cells. The main goal is to find a safe dose and watch for serious side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- engineered immune cells (B7-H3 CAR T cells and PRAME-targeting T cells)
- What this could lead to
- If it works, this could point toward a new treatment option for children with hard-to-treat embryonal tumors that have come back or not responded to standard therapy.
- What could go wrong
- This is a very early, small Phase 1 trial focused on safety, not yet on effectiveness. The treatment can cause severe side effects like cytokine release syndrome and nerve toxicity. It may not shrink tumors or help patients.
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
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Dec 2044
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 year to 23 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: Recipient Inclusion Criteria for Procurement: * Diagnosis of relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor * Refractory disease, residual detectable disease or relapsed disease following available standard of care therapies with known clinical benefit for their specific tumor type, or unable to receive such therapies due to unacceptable toxicity or contraindication * Measurable or evaluable disease by imaging, as determined following most recent therapy * Age ≥ 1 year and \< 24 years * Weight ≥ 10 kg * No systemic steroid exposure within 1 week of procurement * Karnofsky/Lansky score of ≥ 60 (See Appendix 3) * Participants of childbearing potential or capable of fathering a child must agree to use effective contraceptive measure/s (as described in Appendix 5) during study protocol participation through 6 months following the administration of the CAR-TA T cells * ANC \> 500/µL * ALC \> 1000/µL * Platelet count \> 50,000/uL (level can be achieved with transfusion) * Bilirubin ≤ 2.5 mg/dL * Aspartate aminotransferase (AST)/Alanine transaminase (ALT) ≤ 5x the upper limit of normal for age * Serum creatinine Maximum serum creatinine (mg/dL) Age Male Female 1. to \< 2 years 0.6 0.6 2. to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.2 ≥ 16 years 1.7 1.4 OR Creatinine clearance or glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m for patients with levels above * For FOCBP: Negative pregnancy test * Pulse oximetry of \> 90% on room air * Adequate cardiac function defined as: * Shortening fraction of ≥ 27% by echocardiogram, or * Ejection fraction of \> 50% by echocardiogram or radionuclide angiogram (i.e., MUGA). * No acute neurological toxicity \> grade 1 (with the exception of peripheral sensory neuropathy or controlled seizure disorder on anti-epileptics). * The following time frames must have elapsed between prior therapy completion and apheresis cell collection: * Myelosuppressive chemotherapy/immunomodulatory medications: At least 3 weeks, or 6 weeks if prior nitrosourea. * Hematopoietic growth factors: At least 7 days since the completion of therapy with a growth factor. At least 14 days after receiving pegfilgrastim. * Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen. * Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) since the last dose of a monoclonal antibody or checkpoint inhibitor. * Radiotherapy (XRT): At least 3 weeks since XRT, and at least 6 weeks if radiation involved the CNS or lung fields. Exception: There is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable/evaluable disease outside the radiation port or the site of radiation has documented progression. * Autologous stem cell transplant/infusion: At least 6 weeks from their infusion after an autologous stem cell infusion following myeloablative therapy. Patients who received an autologous stem cell infusion following non-myeloablative therapy do not have a wash-out period; they are eligible once they meet all other eligibility requirements, including recovery from acute side effects. * Investigational agent: at least 28 days since receiving an investigational agent. * Adult participant or the legally authorized representative (LAR) of a minor (defined as \<18 years of age) must be capable of providing informed consent. When appropriate, pediatric participants ≥7 years of age will participate in an age-appropriate discussion and provide assent, unless an IRB-approved waiver of assent applies and documentation of the participant's eligibility for the waiver is maintained. Recipient Inclusion Criteria for CAR-TA T cell product Infusion: * No systemic steroid exposure within 1 week prior to protocol therapy initiation * Karnofsky/Lansky score of ≥ 60 * ANC \> 750/uL * Platelet count \> 75,000/uL * Bilirubin ≤ 2.5 mg/dL * AST/ALT ≤ 5x the upper limit of normal for age * Serum creatinine Maximum serum creatinine (mg/dL) Age Male Female 1 to \< 2 years 0.6 0.6 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.2 ≥ 16 years 1.7 1.4 OR Creatinine clearance or glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m for patients with levels above * For FOCBP: Negative pregnancy test * Participants of childbearing potential or capable of fathering a child must agree to use effective contraceptive measure/s through 6 months following the administration of the CAR-TA T cells * Adequate respiratory function defined as oxygen saturation 90% or higher on room air * For participants who underwent prior mediastinum-directed therapies (e.g., post radiation to chest): resolution of any respiratory symptoms * Adequate respiratory rate, defined as \<30 breaths per minute for patients aged \<18 years, and \<25 breaths per minute for patients aged ≥18 years (respiratory rate may be repeated if initial value is thought to be temporarily abnormal; if repeated, 2 consecutive readings obtained ≥30 minutes apart must be adequate to be eligible) * No acute neurological toxicity \> grade 1 (with the exception of peripheral sensory neuropathy or controlled seizure disorder on anti-epileptics). * Adequate cardiac function defined as: * Shortening fraction of ≥ 27% by echocardiogram, or * Ejection fraction of \> 50% by echocardiogram or radionuclide angiogram * The following time frames must have elapsed between completion of prior therapy and the initiation of SABRE protocol therapy: * Myelosuppressive chemotherapy: At least 2 weeks from last dose of chemotherapy. * Hematopoietic growth factors: At least 7 days since the completion of therapy with a growth factor. At least 14 days after receiving pegfilgrastim. * Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen. * Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) since the last dose of a monoclonal antibody or checkpoint inhibitor. * Radiotherapy (XRT): At least 3 weeks since XRT, and at least 6 weeks if radiation involved CNS or lung fields. Exception: There is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable/evaluable disease outside the radiation port or the site of radiation has documented progression. * Investigational agent: At least 28 days since receiving an investigational agent. * Adult participant or the legally authorized representative (LAR) of a minor (defined as \<18 years of age) must be capable of providing informed consent. When appropriate, pediatric participants ≥7 years of age will participate in an age-appropriate discussion and provide assent, unless an IRB-approved waiver of assent applies and documentation of the participant's eligibility for the waiver is maintained. Exclusion Criteria: Recipient Procurement Exclusion Criteria: * Patients with known CNS disease. * Patients with uncontrolled infection/s or known HIV infection * Pregnant or lactating females. * Patients who have undergone previous allogeneic stem cell transplant. * Inability to tolerate leukapheresis (including any contraindication to the use of Anticoagulant Citrate Dextrose solution). Recipient Exclusion Criteria for CAR-TA T cell product Infusions: * Patients with uncontrolled infections or known HIV infection. * Pregnant or lactating females * Whole lung/mediastinal radiation within 12 weeks * Clinically significant systemic illness or medical condition likely to interfere with assessment of safety or efficacy * Patients who have received any live vaccine in the six weeks prior to planned initiation of lymphodepletion * Any contraindication to lymphodepletion or to the use of Cyclophosphamide or Fludarabine, as per the clinical judgment of the PI or treating Sub-I * History of allergy or hypersensitivity to study product excipients (e.g., DMSO)
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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.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Childrens National Hospital
NOT_YET_RECRUITINGWashington D.C., District of Columbia, 20010, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A pill that blocks RAS: can it help children whose tumors came back?
- Can a new drug tame ewing sarcoma when others fail?
- Can a drug duo outsmart resistant tumors?
- Can an antibody drug outsmart resistant Ewing's sarcoma?
- Can a Two-Drug combo outsmart returning childhood cancers?
- Can a Two-Drug combo beat tough childhood cancers?