RhabdomyosarcomaPhase 1SABRENCT07172958
Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)
A Phase 1 study testing Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells in people with Rhabdomyosarcoma and Ewing Sarcoma, run by Children's National Research Institute at 2 sites in United States.
Trial summary
This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and… Read more about this study
- Who is it for?
- Ages 1 year to 23 years
- Conditions the study is for
- Rhabdomyosarcoma, Ewing Sarcoma, Neuroblastoma
- What is being tested?
- Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- 27 Jan 2026to Dec 2044
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 23 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
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- 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
- to < 2 years 0.6 0.6
- 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.
Who cannot join
- 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).
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- 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)
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
Fahmida Hoq, MBBS, MS, 202-476-3634, FHOQ@childrensnational.org
Amy Hont, MD
- Not yet recruiting
Childrens National Hospital
Washington D.C., District of Columbia, 20010
Holly Meany, MD, 2024765697, HMeany@childrensnational.org
Holly Meany, MD
About this study
Official title: Selective Antigen Specific dTβRII-expressing T Cells and B7-H3 CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)
This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Patients will be enrolled to one of three planned dose levels with B7-H3 CAR T cell dose determined based on the percentage of B7-H3 transduced cells (B7-H3+ population of cells), and dTBRII-transduced PRAME TA-specific T cell dose based on the total cell population. Both doses will be based on the recipient's body weight and combined in a 1:1 ratio.
The safety of the CAR-TA T cell product will be evaluated and the maximum tolerated dose (MTD) will be determined. The safety endpoint will be assessed by monitoring for dose limiting toxicities for 28 days following CAR-TA T cell administration.
Read the detailed description
This protocol is designed as a phase I dose-escalation study. Procurement phase: During the procurement phase of this protocol, upon SABRE Procurement Consent and procurement eligibility confirmation, participants will undergo a non-mobilized apheresis for collection of mononuclear cells to be used for the CAR-TA T cell product manufacturing.
Treatment phase: Once the CAR-TA T cell products are released and patients are confirmed eligible for CAR-TA T cell product infusion, participants will undergo protocol therapy at participating site(s), consisting of a standard lymphodepleting chemotherapy preparative regimen with fludarabine and cyclophosphamide, followed by intravenous infusion of the combined CAR-TA T cell product. The DNR-TA T cells and B7-H3 CAR T cells will be generated and combined into a final product comprised of the two T cell components combined at a 1:1 ratio.
Patients will be enrolled to one of three CAR-TA T cell product dose levels (dose levels 1, 2 and 3). There are provisions in place to dose de-escalate for safety concerns (dose level -1).
Fludarabine will be administered intravenously once daily over 30 minutes, days -5 through -2 (4 doses in total). The dose of fludarabine will be 30 mg/m2 /day. Cyclophosphamide will be administered intravenously once daily over 30 minutes, days -5 and -4 (2 doses in total). The dose of cyclophosphamide will be 500 mg/m2 /day.
The first 3 patients enrolled on study will be ≥ 12 years of age at enrollment and treated at dose level 1 (1 x 10e6/kg). If no DLTs are observed in this cohort, enrollment at dose levels 2 (3 x 10e6/kg) and 3 (10 x 10e6/kg) will expand to include patients aged ≥ 1 year and < 24 years.
Patients will remain admitted for at least 7 days following the CAR-TA T cell infusion. All infused patients will be followed with weekly visits during the 28-day dose-limiting toxicity monitoring period where they will be clinically assessed, and safety and research blood draws will be performed.
Ideally, patients should not receive other systemic or local cancer-directed therapies for at least 28 days after the CAR-TA T cell infusion.
Participants will be followed closely for 1 year following the CAR-TA T-cell infusion. After 1 year, yearly assessments will be done up to 15 years. The visits will consist of labs and examinations as well as talking to participants about how they are feeling. Participants will be followed for toxicity until the last follow-up post CAR-TA T cell product administration.
This study will be conducted at Children's National Hospital (CNH). Cell culture manipulations will be carried out in the CETI Good Manufacturing Practice (GMP) facility within Children's National Hospital using current standard operating procedures (SOPs).
Up to 18 toxicity-evaluable participants will be treated on this protocol to meet the primary objective over an estimated accrual period of 5 years
- Conditions
- Rhabdomyosarcoma, Ewing Sarcoma, Neuroblastoma, Wilms Tumor
- Being tested
- Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells
- Keywords
- CAR T Therapy for Embryonal tumors, T cell Therapy for Embryonal tumors
- Medical subject headings
- Rhabdomyosarcoma, Sarcoma, Ewing, Neuroblastoma, Wilms Tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cellsBiological
Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells
| Study group | Role | What they receive |
|---|---|---|
| This is single arm study. | Experimental | Lymphodepleting chemotherapy regimen with cyclophosphamide and fludarabine will be administered prior to CAR-TA T cell product infusion. The DNR-TA T cells and B7-H3 CAR T cells will be generated and combined into a final product comprised of the two T cell components combined at a 1:1 ratio. Receives: Biological: Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the safety of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
The safety endpoint will be assessed by monitoring for dose limiting toxicities (DLT) for 28 days following CAR-TA T cell investigational product administration.
Measured: Within 28 days from the CAR-TA T cell infusion
To determine the manufacturing feasibility of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Manufacturing feasibility will be determined by the number of CAR-TA T cell products produced in sufficient quantities to meet the participant's assigned dose level for at least one infusion, with all product release testing criteria met.
Measured: Within 28 days from the CAR-TA T cell infusion
To determine the clinical feasibility of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Clinical feasibility will be determined by the number of participants with a released product who are eligible and receive at least 1 infusion.
Measured: Within 28 days from the CAR-TA T cell infusion
Secondary outcomes (11)
Determine number of patients who respond to CAR-TA T cell therapy for treatment of diseases under study
To determine the number of patients who respond to CAR-TA T cell therapy for treatment of relapsed or refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor as defined by those that achieve a complete response, partial response, or stable disease following infusion of the CAR-TA T cell product.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
Overall Survival
To determine overall survival at 3-, 6-, and 12-months following CAR-TA T cell infusion.
Measured: Within 12 months of CAR-TA T cell infusion.
To characterize the in vivo immune milieu pre- and post-infusion of CAR-TA T cell product.
To measure levels of circulating cytokines all assessed pre-infusion and post-infusion.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize transgene transduction efficiency (B7-H3 CAR and dTBRII) of the CAR-TA T cell product generated prior to infusion.
To characterize the CAR-TA T cell products generated prior to infusion in terms of transduction efficiency (B7-H3 CAR and dTBRII), with correlation to clinical and immune response.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize reconstitution of anti-tumor immunity following infusion.
Reconstitution of anti-tumor immunity, with evaluation of antigen spreading as an indicator of anti-tumor immunity, and evaluation of patient sample specificity to targeted and non-targeted tumor-associated antigens pre-infusion and post infusion.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
To determine in vivo persistence of infused DNR-TA T cells and B7-H3 CAR T cells at 1-,3-, 6-, and 12-months following infusion of CAR-TA T cell product and evaluate the association with clinical response
To determine persistence of infused DNR-TA T cells and B7-H3 CAR T cells at months 1, 3, 6, and 12 post-infusion and correlate with clinical response.
Measured: Within 12 months of CAR-TA T cell infusion
To characterize the in vivo immune milieu pre- and post-infusion of CAR-TA T cell product.
To measure proportions of in vivo circulating lymphocytes all assessed pre-infusion and post-infusion.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize the in vivo immune milieu pre- and post-infusion of CAR-TA T cell product.
To measure markers of activation and exhaustion all assessed pre-infusion and post-infusion.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize PRAME specificity of the CAR-TA T cell product generated prior to infusion.
To characterize the CAR-TA T cell products generated prior to infusion in terms of PRAME specificity with correlation to clinical and immune response.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize phenotype of the CAR-TA T cell product generated prior to infusion.
To characterize the CAR-TA T cell products generated prior to infusion in terms of phenotype with correlation to clinical and immune response.
Measured: Within 15 years of infusion of CAR-TA T cell therapy.
Progression
To determine progression-free survival at 3-, 6-, and 12- months following CAR-TA T cell infusion.
Measured: Within 12 months of CAR-TA T cell infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2025
- Study start27 Jan 2026
- Primary completionDec 2041 (estimated)
- Study completionDec 2044 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RhabdomyosarcomaStudies of the same condition, open and under way.
- More trials run by Children's National Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI), Cancer Research UK
- Responsible party
- Principal investigator, HMeany, Children's National Research Institute
- Investigators
- Holly Meany, MD, Principal investigator, Children's National Research InstituteAmy Hont, MD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07172958
- Sponsor's protocol ID
- STUDY00001593
- Acronym
- SABRE
- Status last verified
- Aug 2026
- First submitted
- 8 Sep 2025
- Last updated
- 13 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Selective Antigen Specific dTβRII-expressing T Cells and B7-H3 CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)
- ClinicalTrials.gov ID
- NCT07172958
- Acronym / sponsor ID
- SABRE / STUDY00001593
- Conditions
- Rhabdomyosarcoma, Ewing Sarcoma, Neuroblastoma, Wilms Tumor
- Keywords
- CAR T Therapy for Embryonal tumors, T cell Therapy for Embryonal tumors
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), Cancer Research UK
- Study start
- 27 Jan 2026 (actual)
- Primary completion
- Dec 2041 (estimated)
- Study completion
- Dec 2044 (estimated)
- First posted
- 15 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Patients will be enrolled to one of three planned dose levels with B7-H3 CAR T cell dose determined based on the percentage of B7-H3 transduced cells (B7-H3+ population of cells), and dTBRII-transduced PRAME TA-specific T cell dose based on the total cell population. Both doses will be based on the recipient's body weight and combined in a 1:1 ratio.
The safety of the CAR-TA T cell product will be evaluated and the maximum tolerated dose (MTD) will be determined. The safety endpoint will be assessed by monitoring for dose limiting toxicities for 28 days following CAR-TA T cell administration.
Detailed description
This protocol is designed as a phase I dose-escalation study. Procurement phase: During the procurement phase of this protocol, upon SABRE Procurement Consent and procurement eligibility confirmation, participants will undergo a non-mobilized apheresis for collection of mononuclear cells to be used for the CAR-TA T cell product manufacturing.
Treatment phase: Once the CAR-TA T cell products are released and patients are confirmed eligible for CAR-TA T cell product infusion, participants will undergo protocol therapy at participating site(s), consisting of a standard lymphodepleting chemotherapy preparative regimen with fludarabine and cyclophosphamide, followed by intravenous infusion of the combined CAR-TA T cell product. The DNR-TA T cells and B7-H3 CAR T cells will be generated and combined into a final product comprised of the two T cell components combined at a 1:1 ratio.
Patients will be enrolled to one of three CAR-TA T cell product dose levels (dose levels 1, 2 and 3). There are provisions in place to dose de-escalate for safety concerns (dose level -1).
Fludarabine will be administered intravenously once daily over 30 minutes, days -5 through -2 (4 doses in total). The dose of fludarabine will be 30 mg/m2 /day. Cyclophosphamide will be administered intravenously once daily over 30 minutes, days -5 and -4 (2 doses in total). The dose of cyclophosphamide will be 500 mg/m2 /day.
The first 3 patients enrolled on study will be ≥ 12 years of age at enrollment and treated at dose level 1 (1 x 10e6/kg). If no DLTs are observed in this cohort, enrollment at dose levels 2 (3 x 10e6/kg) and 3 (10 x 10e6/kg) will expand to include patients aged ≥ 1 year and < 24 years.
Patients will remain admitted for at least 7 days following the CAR-TA T cell infusion. All infused patients will be followed with weekly visits during the 28-day dose-limiting toxicity monitoring period where they will be clinically assessed, and safety and research blood draws will be performed.
Ideally, patients should not receive other systemic or local cancer-directed therapies for at least 28 days after the CAR-TA T cell infusion.
Participants will be followed closely for 1 year following the CAR-TA T-cell infusion. After 1 year, yearly assessments will be done up to 15 years. The visits will consist of labs and examinations as well as talking to participants about how they are feeling. Participants will be followed for toxicity until the last follow-up post CAR-TA T cell product administration.
This study will be conducted at Children's National Hospital (CNH). Cell culture manipulations will be carried out in the CETI Good Manufacturing Practice (GMP) facility within Children's National Hospital using current standard operating procedures (SOPs).
Up to 18 toxicity-evaluable participants will be treated on this protocol to meet the primary objective over an estimated accrual period of 5 years
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| This is single arm study. | Experimental | Biological: Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cellsLymphodepleting chemotherapy regimen with cyclophosphamide and fludarabine will be administered prior to CAR-TA T cell product infusion. The DNR-TA T cells and B7-H3 CAR T cells will be generated and combined into a final product comprised of the two T cell components combined at a 1:1 ratio. |
Who may qualify
- Ages
- 1 year to 23 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (61 listed)
- 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
Key exclusion criteria (14 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cellsSelective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells | Biological | This is single arm study. |
Primary outcome
To determine the safety of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Time frame: Within 28 days from the CAR-TA T cell infusion
The safety endpoint will be assessed by monitoring for dose limiting toxicities (DLT) for 28 days following CAR-TA T cell investigational product administration.
To determine the manufacturing feasibility of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Time frame: Within 28 days from the CAR-TA T cell infusion
Manufacturing feasibility will be determined by the number of CAR-TA T cell products produced in sufficient quantities to meet the participant's assigned dose level for at least one infusion, with all product release testing criteria met.
To determine the clinical feasibility of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.
Time frame: Within 28 days from the CAR-TA T cell infusion
Clinical feasibility will be determined by the number of participants with a released product who are eligible and receive at least 1 infusion.
11 secondary outcomes
Determine number of patients who respond to CAR-TA T cell therapy for treatment of diseases under study
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
Overall Survival
Time frame: Within 12 months of CAR-TA T cell infusion.
To characterize the in vivo immune milieu pre- and post-infusion of CAR-TA T cell product.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize transgene transduction efficiency (B7-H3 CAR and dTBRII) of the CAR-TA T cell product generated prior to infusion.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize reconstitution of anti-tumor immunity following infusion.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
To determine in vivo persistence of infused DNR-TA T cells and B7-H3 CAR T cells at 1-,3-, 6-, and 12-months following infusion of CAR-TA T cell product and evaluate the association with clinical response
Time frame: Within 12 months of CAR-TA T cell infusion
To characterize the in vivo immune milieu pre- and post-infusion of CAR-TA T cell product.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize the in vivo immune milieu pre- and post-infusion of CAR-TA T cell product.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize PRAME specificity of the CAR-TA T cell product generated prior to infusion.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
To characterize phenotype of the CAR-TA T cell product generated prior to infusion.
Time frame: Within 15 years of infusion of CAR-TA T cell therapy.
Progression
Time frame: Within 12 months of CAR-TA T cell infusion.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2026.
- Individual participant dataSharing planned: No
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Phase I Trial to Determine the Dose and Evaluate the PK and Safety of Lutetium Lu 177 Edotreotide Therapy in Pediatric Participants With SSTR-positive Tumors
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Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
RecruitingObservational study
International PPB/DICER1 Registry
Pleuropulmonary Blastoma, Sertoli-Leydig Cell Tumor
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Holly Meany, MD principal investigator
Children's National Research Institute
Amy Hont, MD principal investigator
Children's National Research Institute
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells in people with Rhabdomyosarcoma and Ewing Sarcoma, run by Children's National Research Institute at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 61 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Holly Meany, MD (2024765697, HMeany@childrensnational.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the safety of autologous CAR-TA T cells following LD chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor. (time frame: Within 28 days from the CAR-TA T cell infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2026.