CD123 Redirected T Cells for AML in Pediatric Subjects

Eligibility Criteria

• 1. Male and female patients ≥ 1 and ≤ 29 years of age at time of consent.
• 2. AML in second or greater relapse, post-transplant relapse, or chemotherapy-refractory disease. Specifically:
• 1. Second or greater relapse defined as flow cytometric confirmation of myeloid leukemia of at least 0.1% after second documented complete remission; OR
• 2. Any detectable disease post-allogeneic transplant with flow cytometric confirmation (MRD) of myeloid leukemia of at least 0.1% (as confirmed by Hematologics); OR
• 3. Refractory disease, defined as persistent bone marrow involvement with \>5% blasts after two courses of induction chemotherapy for patients at initial presentation or \>5% bone marrow blasts after one course of re-induction chemotherapy for patients who have relapsed after previously achieving a CR.
• 3. Subjects must have a suitable stem cell donor identified with projected ability to proceed to transplant within 6-8 weeks of CART123 infusion.
• 4. Adequate organ function defined as:
• 1. A serum creatinine based on age/gender
• 2. Adequate liver function
• 5. Adequate performance status defined as Lansky or Karnofsky score ≥ 50
• 6. Signed informed consent must be obtained.
• 7. No contraindications for leukapheresis (unless apheresis product previously acquired).
• 8. Subjects of reproductive potential must agree to use acceptable birth control methods.
• 1. Pregnant or lactating (nursing) women.
• 2. Patients with relapsed AML with t(15:17).
• 3. Patients \< 6 months from alloHSCT.
• 4. HIV infection.
• 5. Active hepatitis B or hepatitis C infection.
• 6. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy
• 7. Patients requiring chronic treatment with systemic steroids or immunosuppressant medications. Low-dose physiologic replacement therapy with corticosteroids, topical steroids and inhaled steroids are acceptable. For additional details regarding use of steroids and immunosuppressant medications (including washout requirements prior to CAR T cell administration).
• 8. Any uncontrolled active medical disorder that would preclude participation as outlined.
• 9. Uncontrolled active infection
• 10. Subjects with CNS3 disease that is progressive on therapy or with CNS parenchymal lesions that may increase the risk of CNS toxicity. Subjects with adequately treated CNS leukemia are eligible.
• 11. Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).
• 12. Patients with any prior history of myeloproliferative neoplasm.
• 13. Patients with somatic JAK2 V617F mutation by PCR or next generation sequencing.