Pediatric Solid TumorPhase 1NCT04897321
B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)
A Phase 1 study testing Fludarabine, Cyclophosphamide and MESNA in people with Pediatric Solid Tumor and Osteosarcoma, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
3CAR is being done to investigate an immunotherapy for patients with solid tumors. It is a Phase I clinical trial evaluating the use of autologous T cells genetically engineered to express B7-H3-CARs for patients ≤ 21 years old, with relapsed/refractory B7-H3+ solid tumors. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Pediatric Solid Tumor, Osteosarcoma, Rhabdomyosarcoma
- What is being tested?
- Fludarabine, Cyclophosphamide and MESNA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Jul 2022to 1 Mar 2028
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Procurement and T-cell production eligibility
- a previously collected, autologous leukapheresis product can be used for T-cell production
- Age ≤21 years old
- B7-H3+ solid tumor with measurable disease; B7-H3 expression will be evaluated by standard immunohistochemistry (IHC) using a previously obtained biopsy; a tumor is considered B7-H3 positive with an H-score ≥100
- Estimated life expectancy of >12 weeks
- Karnofsky or Lansky (age-dependent) performance score ≥50
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- For females of child bearing age:
- Not pregnant with negative serum pregnancy test within 7 days prior to enrollment
- Not lactating with intent to breastfeed
- Meets eligibility criteria to undergo autologous apheresis, or have previously undergone autologous apheresis
- Treatment eligibility
- Age ≤21 years old
- B7-H3+ solid tumor with measurable disease
- Evidence of relapsed or refractory disease after standard first-line therapy
- Estimated life expectancy of >8 weeks
- Karnofsky or Lansky (age-dependent) performance score≥50
- Echocardiogram with a ventricular ejection fraction
- >40%; or shortening fraction ≥25%
- Adequate renal function defined as creatinine clearance or radioisotope GFR 50 ml/min/1.73m2 (GFR 40 ml/min/1.73m2 if < 2 years of age)
- Adequate pulmonary function defined as pulse oximetry ≥92% on room air or forced vital capacity (FVC) ≥50% of predicted value
- Total Bilirubin ≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤5 times the upper limit of normal for age
- Hemoglobin≥ 7g/dL (can be transfused)
- Platelet count >50,000/uL (can be transfused)
- Absolute neutrophil count (ANC) ≥ 1000/uL
- Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy
- For females of child bearing age:
- Not pregnant with negative serum pregnancy test within 7 days prior to enrollment
- Not lactating with intent to breastfeed
- If sexually active, agreement to use birth control until 3 months after T-cell infusion. Male partners should use a condom.
- Available autologous transduced T-cell product that has met GMP release criteria
- Agreement to participate in long-term follow-up protocol for patients, who have received genetically modified cell products
Who cannot join
- Known primary immunodeficiency
- Known HIV positivity
- Severe intercurrent bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection)
- History of hypersensitivity reactions to murine protein-containing products
- Rapidly progressive disease (in the opinion of the study PIs)
- Known primary immunodeficiency
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- History of HIV infection
- Severe, uncontrolled intercurrent bacterial, viral or fungal infection
- History of hypersensitivity reactions to murine protein-containing products
- Receiving systemic steroid therapy exceeding the equivalent of 0.5 mg/kg/day of methylprednisolone, in the 7 days prior to B7-H3-CAR T-cell infusion
- Receiving systemic therapy in the 14 days prior to CAR T-cell infusion, which will interfere with the activity of the B7-H3-CAR product (in the opinion of the study PIs).
- Rapidly progressing disease (in the opinion of the study PIs)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Chris DeRenzo, MD, 888-226-4343, referralinfo@stjude.org
Chris DeRenzo, MD
About this study
3CAR is being done to investigate an immunotherapy for patients with solid tumors. It is a Phase I clinical trial evaluating the use of autologous T cells genetically engineered to express B7-H3-CARs for patients ≤ 21 years old, with relapsed/refractory B7-H3+ solid tumors. This study will evaluate the safety and maximum tolerated dose of B7-H3-CAR T cells.The purpose of this study is to find the maximum (highest) dose of B7-H3-CAR T cells that are safe to give to patients with B7-H3-positive solid tumors.
Primary objective
Read more
To determine the safety of one intravenous infusion of autologous, B7-H3-CAR T cells in patients (≤ 21 years) with recurrent/refractory B7-H3+ solid tumors after lymphodepleting chemotherapy
Secondary objective
To evaluate the antitumor activity of B7-H3-CAR T cells
Exploratory objectives
- To evaluate the tumor environment after treatment with B7-H3-CAR T cells
- To assess the immunophenotype, clonal structure and endogenous repertoire of B7-H3-CAR T cells and unmodified T cells
- To characterize the cytokine profile in the peripheral blood after treatment with B7-H3-CAR T cells
Read the detailed description
Treatment will include a single infusion of B7-H3-CAR T cells after lymphodepleting chemotherapy, with dosing based on the number of CAR+ T cells and patient weight. The study will evaluate the safety and maximum tolerated dose (MTD) of B7-H3-CAR T cells, using a standard 3+3 study design and a 6-week evaluation period. The total study duration will be 1 year, at which point patients will enroll on our existing institutional long-term follow-up protocol.
- Conditions
- Pediatric Solid Tumor, Osteosarcoma, Rhabdomyosarcoma, Neuroblastoma, Ewing Sarcoma, Wilms Tumor
- Being tested
- Fludarabine, Cyclophosphamide, MESNA, B7-H3 CAR T cells
- Medical subject headings
- Osteosarcoma, Rhabdomyosarcoma, Neuroblastoma, Sarcoma, Ewing, Wilms Tumor, Adrenal Cortex Neoplasms, Desmoplastic Small Round Cell Tumor, Neoplasms, Germ Cell and Embryonal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara
Fludarabine phosphate is a synthetic purine nucleoside analog. It acts by inhibiting DNA polymerase, ribonucleotide reductase and DNA primase by competing with the physiologic substrate, deoxyadenosine triphosphate, resulting in inhibition of DNA synthesis. Intravenous
CyclophosphamideDrug
Also called Cytoxan
Cyclophosphamide is a nitrogen mustard derivative. It acts as an alkylating agent that causes cross-linking of DNA strands by binding with nucleic acids and other intracellular structures, thus interfering with the normal function of DNA. Intravenous
MESNADrug
Also called Mesnex
Mesna is a synthetic sulfhydryl (thiol) compound. Mesna contains free sulfhydryl groups that interact chemically with urotoxic metabolites of oxaza-phosphorine derivatives such as cyclophosphamide and ifosfamide
B7-H3 CAR T cellsDrug
Also called CAR T- cell infusion
The study participant will receive B7-H3-CAR T cells by vein, through either an IV or a central line.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Phase | Other | During the treatment phase, the participant receives an infusion of the B7-H3-CAR T cells that were made in the Collection and Manufacturing Phase. Chemotherapy is given for several days prior to the cellular infusion. Patients are then monitored for possible side effects, as well as effects of the treatment on their cancer. Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Drug: MESNA; Drug: B7-H3 CAR T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of B7-H3-CAR T cells
A phase I design to determine the maximum tolerated dose (MTD) of autologous, B7-H3-CAR T cells. Four dose levels (3x10^5/kg, 1x10^6/kg, 3x10^6/kg, and 1x10^7/kg) will be evaluated.
Measured: 6 weeks after B7-H3-CAR T cell infusion
Secondary outcomes (1)
Clinical Response
The number of patients with objective responses (complete response (CR) + partial response (PR)) determined by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.
Measured: 6 weeks after B7-H3-CAR 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 posted21 May 2021
- Study start6 Jul 2022
- Primary completion1 Mar 2027 (estimated)
- Study completion1 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Solid TumorStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Chris DeRenzo, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04897321
- Sponsor's protocol ID
- 3CAR
- Other IDs
- NCI-2021-10933
- Status last verified
- May 2026
- First submitted
- 11 May 2021
- Last updated
- 19 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)
- ClinicalTrials.gov ID
- NCT04897321
- Acronym / sponsor ID
- 3CAR
- Conditions
- Pediatric Solid Tumor, Osteosarcoma, Rhabdomyosarcoma, Neuroblastoma, Ewing Sarcoma, Wilms Tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 6 Jul 2022 (actual)
- Primary completion
- 1 Mar 2027 (estimated)
- Study completion
- 1 Mar 2028 (estimated)
- First posted
- 21 May 2021 (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.
3CAR is being done to investigate an immunotherapy for patients with solid tumors. It is a Phase I clinical trial evaluating the use of autologous T cells genetically engineered to express B7-H3-CARs for patients ≤ 21 years old, with relapsed/refractory B7-H3+ solid tumors. This study will evaluate the safety and maximum tolerated dose of B7-H3-CAR T cells.The purpose of this study is to find the maximum (highest) dose of B7-H3-CAR T cells that are safe to give to patients with B7-H3-positive solid tumors.
Primary objective
To determine the safety of one intravenous infusion of autologous, B7-H3-CAR T cells in patients (≤ 21 years) with recurrent/refractory B7-H3+ solid tumors after lymphodepleting chemotherapy
Secondary objective
To evaluate the antitumor activity of B7-H3-CAR T cells
Exploratory objectives
- To evaluate the tumor environment after treatment with B7-H3-CAR T cells
- To assess the immunophenotype, clonal structure and endogenous repertoire of B7-H3-CAR T cells and unmodified T cells
- To characterize the cytokine profile in the peripheral blood after treatment with B7-H3-CAR T cells
Detailed description
Treatment will include a single infusion of B7-H3-CAR T cells after lymphodepleting chemotherapy, with dosing based on the number of CAR+ T cells and patient weight. The study will evaluate the safety and maximum tolerated dose (MTD) of B7-H3-CAR T cells, using a standard 3+3 study design and a 6-week evaluation period. The total study duration will be 1 year, at which point patients will enroll on our existing institutional long-term follow-up protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Phase | Other | Drug: Fludarabine, Drug: Cyclophosphamide, Drug: MESNA, Drug: B7-H3 CAR T cellsDuring the treatment phase, the participant receives an infusion of the B7-H3-CAR T cells that were made in the Collection and Manufacturing Phase. Chemotherapy is given for several days prior to the cellular infusion. Patients are then monitored for possible side effects, as well as effects of the treatment on their cancer. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (32 listed)
- Procurement and T-cell production eligibility
- a previously collected, autologous leukapheresis product can be used for T-cell production
- Age ≤21 years old
- B7-H3+ solid tumor with measurable disease; B7-H3 expression will be evaluated by standard immunohistochemistry (IHC) using a previously obtained biopsy; a tumor is considered B7-H3 positive with an H-score ≥100
- Estimated life expectancy of >12 weeks
Key exclusion criteria (12 listed)
- Known primary immunodeficiency
- Known HIV positivity
- Severe intercurrent bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection)
- History of hypersensitivity reactions to murine protein-containing products
- Rapidly progressive disease (in the opinion of the study PIs)
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 |
|---|---|---|
| FludarabineAlso: FludaraFludarabine phosphate is a synthetic purine nucleoside analog. It acts by inhibiting DNA polymerase, ribonucleotide reductase and DNA primase by competing with the physiologic substrate, deoxyadenosine triphosphate, resulting in inhibition of DNA synthesis. Intravenous | Drug | Treatment Phase |
| CyclophosphamideAlso: CytoxanCyclophosphamide is a nitrogen mustard derivative. It acts as an alkylating agent that causes cross-linking of DNA strands by binding with nucleic acids and other intracellular structures, thus interfering with the normal function of DNA. Intravenous | Drug | Treatment Phase |
| MESNAAlso: MesnexMesna is a synthetic sulfhydryl (thiol) compound. Mesna contains free sulfhydryl groups that interact chemically with urotoxic metabolites of oxaza-phosphorine derivatives such as cyclophosphamide and ifosfamide | Drug | Treatment Phase |
| B7-H3 CAR T cellsAlso: CAR T- cell infusionThe study participant will receive B7-H3-CAR T cells by vein, through either an IV or a central line. | Drug | Treatment Phase |
Primary outcome
Safety of B7-H3-CAR T cells
Time frame: 6 weeks after B7-H3-CAR T cell infusion
A phase I design to determine the maximum tolerated dose (MTD) of autologous, B7-H3-CAR T cells. Four dose levels (3x10^5/kg, 1x10^6/kg, 3x10^6/kg, and 1x10^7/kg) will be evaluated.
1 secondary outcome
Clinical Response
Time frame: 6 weeks after B7-H3-CAR 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 19 May 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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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.
Chris DeRenzo, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fludarabine, Cyclophosphamide and MESNA in people with Pediatric Solid Tumor and Osteosarcoma, run by St. Jude Children's Research Hospital at 1 site 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 32 inclusion and 12 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 19 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 Chris DeRenzo, MD (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of B7-H3-CAR T cells (time frame: 6 weeks after B7-H3-CAR T cell infusion). 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 19 May 2026; AzTrials last refreshed its copy on 6 Nov 2026.