CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies

Valentín Ortíz-Maldonado, Susana Rives, Maria Castellà, Anna Alonso-Saladrigues, Daniel Benítez-Ribas, Miguel Caballero-Baños, Tycho Baumann, Joan Cid, Enric Garcia-Rey, Cristina Llanos, Montserrat Torrebadell, Neus Villamor, Eva Giné, Marina Díaz-Beyá, Laia Guardia, Mercedes Montoro, Albert Català, Anna Faura, E. Azucena González, Marta Español-RegoNela Klein-González, Laia Alsina, Pedro Castro, Iolanda Jordan, Sara Fernández, Federico Ramos, Guillermo Suñé, Unai Perpiñá, Josep M. Canals, Miquel Lozano, Esteve Trias, Andrea Scalise, Sara Varea, Joaquín Sáez-Peñataro, Ferran Torres, Gonzalo Calvo, Jordi Esteve, Álvaro Urbano-Ispizua, Manel Juan, Julio Delgado*

*Corresponding author for this work

Research output: Contribution to journalArticleResearchpeer-review

Abstract

We evaluated the administration of ARI-0001 cells (chimeric antigen receptor T cells targeting CD19) in adult and pediatric patients with relapsed/refractory CD19+ malignancies. Patients received cyclophosphamide and fludarabine followed by ARI-0001 cells at a dose of 0.4–5 × 106 ARI-0001 cells/kg, initially as a single dose and later split into 3 fractions (10%, 30%, and 60%) with full administration depending on the absence of cytokine release syndrome (CRS). 58 patients were included, of which 47 received therapy: 38 with acute lymphoblastic leukemia (ALL), 8 with non-Hodgkin's lymphoma, and 1 with chronic lymphocytic leukemia. In patients with ALL, grade ≥3 CRS was observed in 13.2% (26.7% before versus 4.3% after the amendment), grade ≥3 neurotoxicity was observed in 2.6%, and the procedure-related mortality was 7.9% at day +100, with no procedure-related deaths after the amendment. The measurable residual disease-negative complete response rate was 71.1% at day +100. Progression-free survival was 47% (95% IC 27%–67%) at 1 year: 51.3% before versus 39.5% after the amendment. Overall survival was 68.6% (95% IC 49.2%–88%) at 1 year. In conclusion, the administration of ARI-0001 cells provided safety and efficacy results that are comparable with other academic or commercially available products. This trial was registered as ClinicalTrials.gov: NCT03144583.

Original languageAmerican English
JournalMolecular Therapy
DOIs
Publication statusAccepted in press - 2020

Keywords

  • A3B1
  • ALL
  • ARI-0001
  • CART-cells
  • CD19
  • NHL

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