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Haematological malignancies

1314 - ZUMA-4 preliminary results: phase 1 study of KTE-C19 chimeric antigen receptor T cell therapy in pediatric and adolescent patients (pts) with relapsed/refractory acute lymphoblastic leukemia (R/R ALL)


11 Sep 2017


Haematological malignancies


Cancers in Adolescents and Young Adults (AYA);  Leukaemia


Daniel Lee


Annals of Oncology (2017) 28 (suppl_5): v355-v371. 10.1093/annonc/mdx373


D.W. Lee1, A.S. Wayne2, V. Huynh3, R. Handgretinger4, R. Pieters5, G. Michele6, A. Baruchel7, T. Feuchtinger8, Y. Bertrand9, M. Hemiston10, P.A. Brown11, J. Rossi12, Y. Jiang13, L. Navale13, S. Stout14, J. Aycock14, A. Mardiros12, J. Wiezorek14, R. Jain14

Author affiliations

  • 1 Pediatric Stem Cell Transplant Division Of Pediatric Hematology/oncology, University of Virginia, 22908 - Charlottesville/US
  • 2 Children's Center For Cancer And Blood Diseases, Children's Hospital Los Angeles, Usc-norris Comprehensive Cancer Center, Keck School Of Medicine, University of Southern California, Los Angeles/US
  • 3 Department Of Epidemiology And Chao Family Comprehensive Cancer Center/ Hyundai Cancer Institute, Children's Hospital of Orange County, Orange/US
  • 4 Universität Children's Hospital, Eberhard Karls Universität Tübingen, Tübingen/DE
  • 5 Pediatric Oncology, Princess Maxima Center for Pediatric Oncology, Utrecht/NL
  • 6 Hôpital De La Timone, Centre Hospitalier, Universitaire, Marseille Universite, Marseille/FR
  • 7 Pediatric Hematology Hôpital Robert Debré, CHU Paris, Paris/FR
  • 8 Dr. Von Hauner University Children’s Hospital, Ludwig-Maximilians-University Munich, Munich/DE
  • 9 Department Of Pediatric Hematology And Immunology, Centre Hospitalier Universitaire, Lyon/FR
  • 10 Helen Diller Family Comprehensive Cancer Center, University of California at San Francisco, San Francisco/US
  • 11 Pediatric Leukemia Program, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore/US
  • 12 Translational Sciences, Kite Pharma, Santa Monica/US
  • 13 Biostatistics, Kite Pharma, Santa Monica/US
  • 14 Clinical Development, Kite Pharma, Santa Monica/US


Abstract 1314


ALL is the most common childhood malignancy; up to 20% of pts relapse after initial therapy, and have poor clinical outcomes (Hoffman and Gore. Front Oncol 2014). Promising results were observed with KTE-C19, an anti-CD19 chimeric antigen receptor (CAR) T cell therapy, in B cell malignancies (Locke at al. AACR 2017, #9986). We present preliminary ZUMA-4 phase 1 results.


Pts aged 2-21 y with R/R ALL and adequate organ function received the planned dose of 1 or 2 × 106 CAR T cells/kg after low-dose conditioning chemotherapy (cyclophosphamide/fludarabine, CyFlu). Pts enrolled after the study’s dose-limiting toxicities (DLTs) portion receive tocilizumab ≤36 h after CAR T cell infusion. Phase 1 primary endpoint is the incidence of DLTs. Secondary endpoints include efficacy and biomarker assessments.


As of Dec 31, 2016, 4 of 5 enrolled pts have been treated; median follow-up, 5.3 mo (1.9-8.6). All pts had ≥2 prior lines of therapy and 1 pt had prior stem cell transplant (SCT). All pts received bridging chemotherapy due to high disease burden (baseline blasts, 41-99%) prior to KTE-C19. KTE-C19 was successfully manufactured in a centralized 6-7 d process across a range of baseline absolute lymphocyte counts (0.5–17.1 × 109/L) and CD4:CD8 ratios (0.1-0.7). KTE-C19 could not be manufactured for 1 pt who progressed with WBC >150,000/µL at apheresis and


KTE-C19 after low-dose CyFlu was tolerable and appears safe for further analysis in pediatric and adolescent pts with R/R ALL. KTE-C19 can induce deep remissions in heavily pre-treated pts with high disease burden. Based on these results, ZUMA-4 continues to enroll (NCT02625480).

Clinical trial identification


Legal entity responsible for the study

Kite Pharma


Kite Pharma


D.W. Lee: Honoraria from Juno. A.S. Wayne: Pfizer: Consultancy, Honoraria, advisory board; Kite Pharma: Research Funding, Honoraria, Advisory Board; NIH: Patents; MedImmune: Research Funding; Spectrum Pharmaceuticals: Research Funding; Spectrum: Honoraria, advisory board. R. Handgretinger: Patents and Royalties: Milteny Biotech. J. Rossi, Y. Jiang, L. Navale, S. Stout, J. Aycock: Employment/Stock Ownership: Kite Pharma. A. Mardiros: Employment/Stock Ownership: Kite Pharma; Patents: Mustang Bio. J. Wiezorek: Leadership/Employment/Stock Ownership: Kite Pharma. R. Jain: Patent/Employment/Stock Ownership: Kite Pharma. All other authors have declared no conflicts of interest.

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