RecruitingPhase 1NCT04897321

B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)


Sponsor

St. Jude Children's Research Hospital

Enrollment

32 participants

Start Date

Jul 6, 2022

Study Type

INTERVENTIONAL

Conditions

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. 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


Eligibility

Max Age: 21 Years

Inclusion Criteria32

  • 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
  • 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

Exclusion Criteria12

  • 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
  • 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)

Interventions

DRUGFludarabine

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

DRUGCyclophosphamide

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

DRUGMESNA

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

DRUGB7-H3 CAR T cells

The study participant will receive B7-H3-CAR T cells by vein, through either an IV or a central line.


Locations(1)

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

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NCT04897321


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