1. CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies
- Author
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Miquel Lozano, Iolanda Jordan, Enric Garcia-Rey, Manel Juan, Miguel Caballero-Baños, Laia Guardia, Pedro Castro, E. Azucena González, Andrea Scalise, Eva Giné, Jordi Esteve, Ferran Torres, Neus Villamor, Esteve Trias, Alvaro Urbano-Ispizua, Marina Díaz-Beyá, Julio Delgado, Cristina Llanos, Sara Fernández, Unai Perpiñá, Josep M. Canals, Marta Español-Rego, Montserrat Torrebadell, Federico Ramos, Sara Varea, Mercedes Montoro, Tycho Baumann, Joan Cid, Anna Alonso-Saladrigues, M. Castella, Joaquín Sáez-Peñataro, Gonzalo Calvo, Valentín Ortiz-Maldonado, Susana Rives, Daniel Benitez-Ribas, Laia Alsina, Albert Català, Anna Faura, Nela Klein-González, and Guillermo Suñe
- Subjects
medicine.medical_specialty ,Cyclophosphamide ,Chronic lymphocytic leukemia ,Gastroenterology ,Cell therapy ,03 medical and health sciences ,0302 clinical medicine ,Refractory ,Multicenter trial ,Internal medicine ,Drug Discovery ,Genetics ,medicine ,Molecular Biology ,030304 developmental biology ,Pharmacology ,0303 health sciences ,business.industry ,medicine.disease ,Lymphoma ,Fludarabine ,Cytokine release syndrome ,030220 oncology & carcinogenesis ,Molecular Medicine ,business ,medicine.drug - 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 .
- Published
- 2021
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