Safety and Feasibility Study of CD19 Chimeric Antigen Receptor (CAR) T Cells in Children with Relapsed or Refractory CD19 Positive Acute Lymphoblastic Leukemia or Lymphoma
This study seeks to examine the efficacy and safety of the administration of autologous T cells that have been modified through the introduction of a chimeric antigen receptor (CAR) targeting the B cell surface antigen CD19 following administration of chemotherapy lymphodepletion regimen in children with relapsed or refractory acute lymphoblastic leukemia (ALL) or lymphoma. The overall goal of this study is to validate the safety profile of administration CD19-CAR T cells and describe the response rate in children with relapsed/refractory ALL or lymphoma.
• Subjects must have relapsed or refractory ALL or lymphoma treated with at least two lines of therapy. Disease must have either progressed after the last regimen or presented failure to achieve partial or complete remission with the last regimen.
• The patient's disease must be CD19 positive, either by immunohistochemistry or flow cytometry analysis on the last biopsy available.
• Age 1-17 years.
• Performance status: Subjects \> 10 years of age: Karnofsky ≥ 50%; Subjects ≤ 10 years of age: Lansky scale ≥ 50%.
• Normal organ function.
‣ Total bilirubin ≤ 3 times upper limit of normal
⁃ AST (SGOT) ≤ 5 times upper limit of normal
⁃ ALT (SGPT) ≤ 5 times upper limit of normal
⁃ Serum Creatinine ≤ 2 times upper limit of normal
⁃ Subjects must have the following hematologic function parameters: Hemoglobin (Hb) level \> 8 g/dL; Absolute Lymphocyte Count \> 0.1x10\^9/L; Platelet \> 50x10\^9/L
• Prior therapy wash-out. At least 2 weeks or 5 half lives, whichever is shorter, must have elapsed since any prior systemic therapy at the time the subject is planned for leukapheresis.
• Subjects' parent or legal guardian must have the ability to understand and the willingness to sign a written informed consent document.