Genetically Engineered Cells (B7-H3.CD28Z.CART Cells) for the Treatment of Children and Young Adults with Relapsed or Refractory Solid Tumors Expressing B7-H3
This phase I trial studies the side effects and best dose of B7-H3.CD28Z.chimeric antigen receptor (CAR)T cells in treating children and young adults with solid tumors that have come back after a period of improvement (relapsed) or that have not responded to previous treatment (refractory) and have sufficient levels of a protein called B7-H3. B7-H3 is over-expressed on many tumor cells, making it potentially a good target for cancer cell therapy. B7-H3.CD28Z.CART cells are a cellular therapy that involves putting a gene (gene modification) into a patient's own T cells, which are a part of the immune system that usually helps fight infection and prevent/fight cancers. T cells are collected and modified in a specialized laboratory to better recognize and bind to the B7-H3 markers on tumor cells. When the B7-H3 CART cells are re-infused into the bloodstream, they may recognize, bind to, and kill tumor cells. Patients also receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide prior to B7-H3.CD28Z.CART cells, which reduces T-cells and other lymphocytes to prepare the immune system for the CAR T cell infusion. Fludarabine injection is in a class of medications called purine analogs. It works by slowing or stopping the growth of tumor cells in the body. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell’s deoxyribonucleic acid and may kill tumor cells. It may also lower the body’s immune response. Giving B7-H3.CD28Z.CART cells with lymphodepleting chemotherapy may be a safe treatment for children and young adults with relapsed or refractory solid tumors expressing B7-H3.
Inclusion Criteria
- PRESCREENING: Participants must have histologically confirmed diagnosis of a solid tumor that is relapsed or refractory for which standard curative measures do not exist or are no longer effective
- PRESCREENING: Participant must have adequate pre-trial tumor material available to determine B7-H3 status. Tumor tissue from the most recent resection or biopsy of recurrent disease is preferred. If unavailable, tumor tissue from prior recurrences or from the time of initial diagnosis is acceptable
- PRESCREENING: Age >= 9 months and < 30 years
- PRESCREENING: Lansky/Karnofsky performance status >= 50%
- PRESCREENING: Life expectancy of greater than 12 weeks
- PRESCREENING: Participants who are screened for this trial should be reasonably anticipated to meet the eligibility criteria for enrollment described if their tumor is B7-H3- positive
- PRESCREENING: Ability to understand and/or the willingness of their parent or legally authorized representative to sign a written informed consent document for prescreening
- ENROLLMENT: Participants must have histologically confirmed diagnosis of a solid tumor that is relapsed or refractory for which standard curative measures do not exist or are no longer effective
- ENROLLMENT: Participants must have measurable or evaluable disease for dose escalation. For expansion phase, participants with neuroblastoma must have measurable or evaluable disease by International Neuroblastoma Response Criteria (INRC). Participants with other solid tumors must have measurable disease by RECIST1.1 for the expansion phase
- ENROLLMENT: Demonstration of B7-H3 expression with H score > 100 by immunohistochemistry (IHC) is required by IHC performed at Boston Children’s Hospital. Participants may choose to enroll on the prescreening portion, which allows for assessment of B7-H3 expression only, prior to enrollment on the full clinical trial
- ENROLLMENT: Age >= 12 months and < 30 years
- ENROLLMENT: Lansky/Karnofsky performance status >= 50%
- ENROLLMENT: Life expectancy of greater than 12 weeks
- ENROLLMENT: Prior Therapy: * At enrollment these criteria do not apply to participants with available leukapheresis products; however, participants must meet all other eligibility criteria and meet criteria to start lymphodepleting chemotherapy * Participants must have received prior radiation therapy and/or chemotherapy and recovered from all acute treatment-related toxicities of prior therapy prior to entering this study. There is no upper limit to the number of prior therapies allowed. Participants must be: ** At least 1 week post any small port radiation therapy; at least 6 weeks from large field or other substantial bone marrow irradiation (craniospinal, whole abdomen, total lung, total body irradiation, > 50% marrow) ** At least 2 weeks since any prior myelosuppressive chemotherapy ** At least 28 days from other investigational antineoplastic or disease-directed agents ** At least 7 days from most recent myeloid growth factor, at least 14 days must have elapsed after receipt of pegfilgrastim ** At least 7 days from prior biologic antineoplastics, tyrosine kinase inhibitor, targeted agent or metronomic non-myelosuppressive chemotherapy ** At least 21 days or 5 half-lives, whichever is shorter, post any treatment with monoclonal antibodies (including checkpoint inhibitors and bevacizumab) ** At least 7 days from dinutuximab treatment ** At least 8 weeks from prior cellular therapy or vaccine therapy with recovery of associated toxicities. If prior CAR T cells, need documented lack of persistence of prior product ** At least 6 weeks post iodine I 131 metaiodobenzylguanidine (131I-MIBG) therapy or other radioisotope therapy ** At least 6 weeks post autologous stem cell therapy infusion following myeloablative conditioning ** Participants can be eligible after autologous stem cell infusion without myelosuppressive therapy at any time as long as other criteria are met ** At least 12 weeks post allogeneic stem cell transplant with no evidence of graft-versus-host disease (GVHD) or ongoing toxicities
- ENROLLMENT: Corticosteroids at or below physiologic doses (replacement therapy for management of pituitary/adrenal insufficiency) is allowed and/or topical administration (e.g. inhaled or dermatologic) is allowed. Hydrocortisone for blood product premedication is allowed
- ENROLLMENT: Hemoglobin >= 7.0g/dL (must be without transfusions or platelet growth factor within 7 days)
- ENROLLMENT: Absolute neutrophil count >= 750/mcL (must be without transfusions or platelet growth factor within 7 days)
- ENROLLMENT: platelets >= 75,000/mcL (must be without transfusions or platelet growth factor within 7 days)
- ENROLLMENT: Creatinine below normal limits for age OR creatinine clearance (as estimated by Cockcroft Gault Equation for participants >= 18 years old [yo] and Bedside Schwartz for participants < 18yo) >= 70mL/min/1.73m^2 * 6 months to 1 year - Maximum serum creatinine: 0.5mg/dL (male); 0.5mg/dL (female) * 1 to < 2 years - Maximum serum creatinine: 0.6mg/dL (male); 0.6mg/dL (female) * 2 to < 6 years - Maximum serum creatinine: 0.8mg/dL (male); 0.8mg/dL (female) * 6 to < 10 years - Maximum serum creatinine: 1mg/dL (male); 1mg/dL (female) * 10 to < 13 years - Maximum serum creatinine: 1.2mg/dL (male); 1.2mg/dL (female) * 13 to < 16 years - Maximum serum creatinine: 1.5mg/dL (male); 1.4mg/dL (female) * >= 16 years - Maximum serum creatinine: 1.7mg/dL (male); 1.4mg/dL (female)
- ENROLLMENT: Serum alanine aminotransferase/aspartate aminotransferase (ALT/AST) < 3.0 X upper limits of normal (ULN)
- ENROLLMENT: Total bilirubin < 3 X ULN, except in participants with confirmed Gilbert’s syndrome, where direct bilirubin must be < 3 X ULN
- ENROLLMENT: Ejection fraction >= 50% or fractional shortening >= 28%, measured by echocardiography
- ENROLLMENT: No evidence of dyspnea at rest
- ENROLLMENT: No exercise intolerance due to pulmonary insufficiency
- ENROLLMENT: Pulse oximetry > 92% while breathing room air
- ENROLLMENT: Females of childbearing potential must have a negative serum or urine pregnancy test
- ENROLLMENT: The effects of B7-H3.CD28z.CART on the developing human fetus are unknown. For this reason and because other chemotherapeutic agents used in the study are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of the study, and for 1 year after receiving the preparative lymphodepletion regimen or for as long as B7-H3.CD28z.CARTs are detectable in peripheral blood. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, and for 1 year after receiving the preparative lymphodepletion regimen or for as long as B7-H3.CD28z.CARTs are detectable in peripheral blood
- ENROLLMENT: Ability to understand and/or the willingness of their parent or legally authorized representative to sign a written informed consent document. Pediatric patients will be included in the consent discussion as age-appropriate and will provide written informed assent as applicable per institutional standard
Exclusion Criteria
- Participants who are receiving any other investigational agents
- Participants with known current brain metastases or leptomeningeal disease. Prior central nervous system (CNS) metastatic disease is allowable if prior resection and/or radiation occurred at least 8 weeks prior to enrollment, without any intervening CNS metastasis, progression or recurrence, and participants are clinically stable as evidenced by no requirements for corticosteroids, no evolving neurologic deficits, and no progression of residual brain abnormalities
- Participants with any prior immunodeficiency or history of autoimmune disease requiring systemic steroids/immunosuppressive medication/ disease modifying agents within the last 2 years
- Prior solid organ transplant. Prior allogeneic or autologous stem cell transplant is permitted
- Active or uncontrolled viral, bacterial or fungal infection. Participants may be receiving ongoing therapy for controlled infection
- Participants with a known additional malignancy other than non-melanomatous skin cancer or carcinoma in situ, unless not requiring active treatment and stable or disease- free for at least 3 years
- CNS disorder such as cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement that in the judgement of the investigator may impair the ability to evaluate neurotoxicity
- History of severe hypersensitivity reaction to compounds of similar chemical or biologic compositions to any agents used in the study or in the manufacturing of cells
- HIV/hepatitis B virus (HBV)/hepatitis C virus (HCV) infection: Participants are required to be negative for HIV antibody or HIV viral load, negative for hepatitis surface antigen (HbsAg) or viral load and negative for HCV antibody or HCV viral load. These participants are ineligible because of the potential for in vivo retroviral recombination events that could lead to replication-competent gamma-retrovirus. A history of HIV, hepatitis B, or hepatitis C is permitted if the viral load is undetectable per quantitative polymerase chain reaction (PCR) and/or nucleic acid testing
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant women are excluded from this study because the effects of B7-H3.CD28Z.CART cells on the developing fetus are unknown and because chemotherapeutic agents with the potential for teratogenic or abortifacient effects are used in this study. Because there is an unknown but potential risk for adverse events in nursing infants, secondary to treatment of the mother with B7-H3.CD28Z.CART cells and chemotherapy, breastfeeding should be discontinued if the mother is treated with T cells on this study (NOTE: breast milk cannot be stored for future use while the mother is being treated on study)
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT07358260.
Locations matching your search criteria
United States
Massachusetts
Boston
PRIMARY OBJECTIVES:
I. To describe the feasibility of manufacturing autologous T cells transduced with retroviral vector expressing B7- H3.CD28Z.CAR for intravenous administration in children and young adults with relapsed/refractory solid tumors expressing B7-H3.
II. To identify the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of autologous anti-B7-H3-CAR-CD28zeta-expressing T-cells (B7-H3.CD28Z.CART cells) in children and young adults with relapsed/refractory solid tumors expressing B7-H3.
SECONDARY OBJECTIVES:
I. To describe the safety and dose-limiting toxicities of B7-H3.CD28Z.CART cells in children and young adults with relapsed/refractory solid tumors expressing B7-H3.
II. To estimate the objective response rate (ORR) per revised International Neuroblastoma Response Criteria (INRC) in participants with relapsed or refractory high-risk neuroblastoma or per Response Evaluation Criteria in Solid Tumors (RECIST)1.1 in participants with other solid tumors who are treated with B7-H3.CD28Z.CART cells.
III. To estimate the progression free survival (PFS) and overall survival (OS) rates of participants with relapsed or refractory neuroblastoma or other solid tumors who participate in this study and in the subset of participants who are treated with B7-H3.CD28Z.CART cells.
IV. To describe safety and feasibility of a second intravenous B7-H3.CD28Z.CART cell infusion in select participants with relapsed/refractory solid tumors expressing B7-H3.
EXPLORATORY OBJECTIVES:
I. To measure expansion/persistence/phenotype of adoptively transferred B7-H3.CD28Z.CART in the blood and correlate with antitumor effects after CAR T cell infusion.
II. To correlate expansion/persistence and clinical response with the results of B7-H3 immunohistochemistry (IHC).
III. Evaluate whether antigen expression or tumor microenvironment changes are correlated with response to CAR T cells (clinical response, CAR expansion or persistence), when tissue is available.
IV. Conduct analysis of manufactured B7-H3.CD28Z.CART product, as well as baseline and post-infusion immune cell populations to identify biomarkers of CAR T cell activity in blood of participants.
V. Analyze the cytokine inflammatory trajectory in patient peripheral blood at baseline and following CAR T cell administration in relation to CAR T cell expansion and activity.
OUTLINE: This is a dose-escalation study of B7-H3.CD28Z.CART cells followed by a dose-expansion study.
Patients who do not have a prior acceptable leukapheresis product undergo leukapheresis for B7-H3.CD28Z.CART cell manufacturing within 30 days of study enrollment. Patients receive lymphodepleting chemotherapy consisting of fludarabine intravenously (IV) over 30 minutes followed by cyclophosphamide IV over 1 hour on days -4 to -2. Patients then receive B7-H3.CD28Z.CART cells IV on day 0. Patients with a benefit (such as tumor shrinkage) from the first B7-H3.CD28Z.CART cells infusion who did not have unacceptable side effects and are at least 60 days beyond previous infusion may be eligible to receive a second infusion of B7-H3.CD28Z.CART cells after lymphodepleting chemotherapy as above. Patients also undergo echocardiogram (ECHO) and blood sample collection throughout the study. Patients may also undergo computed tomography (CT), magnetic resonance imaging (MRI), positron emission tomography (PET), and/or bone marrow biopsy and aspiration throughout the study.
After completion of study treatment, patients are followed up at 6 weeks, at months 2, 3, 6, 9, 12, 15, 18, 21, and 24, and then annually until year 15.
Trial PhasePhase I
Trial Typetreatment
Lead OrganizationDana-Farber Harvard Cancer Center
Principal InvestigatorNatalie Bucheimer Collins
- Primary ID25-648
- Secondary IDsNCI-2026-06641
- ClinicalTrials.gov IDNCT07358260