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

2020; Elsevier BV; Volume: 29; Issue: 2 Linguagem: Inglês

10.1016/j.ymthe.2020.09.027

1525-0024

Valentín Ortiz‐Maldonado, Susana Rives, María Castellá, Anna Alonso‐Saladrigues, Daniel Benítez‐Ribas, Miguel Caballero‐Baños, Tycho Baumann, Joan Cid, Enric García‐Rey, Cristina Llanos, Montserrat Torrebadell, Neus Villamor, Eva Giné, Marina Díaz‐Beyá, Laia Guardia, Mercedes Montoro, Albert Catalá, Anna Faura, Esther González, Marta Español‐Rego, Nela Klein-González, Laia Alsina, Pedro Castro, Iolanda Jordán, Sara Fernández, Federico Ramos, Guillermo Suñé, Unai Perpiñá, Josep M. Canals, Miquel Lozano, Esteve Trias, Andrea Scalise, Sara Varea, Joaquín Sáez‐Peñataro, Ferrán Torres, Gonzalo Calvo, Jordi Esteve, Álvaro Urbano‐Ispizua, Manel Juan, Julio Delgado,

Neuroblastoma Research and Treatments

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.