{"entity":{"id":"cart19-be-01","kind":"trial","name":"CART19-BE-01 (ARI-0001 CAR-T, Hospital Clínic de Barcelona)","aka":["CART19-BE-01","ARI-0001 pilot study"],"tldr":"The academic trial at Hospital Clínic and Sant Joan de Déu in Barcelona that tested a CAR-T cell therapy made in the hospital itself, and led to Spain authorising it in 2021.","summary":"CART19-BE-01 tested ARI-0001, a CD19-directed CAR-T cell product with 4-1BB and CD3-zeta signalling designed and manufactured at Hospital Clínic de Barcelona, in adults and children with relapsed or refractory CD19-positive leukaemia or lymphoma. Patients received cyclophosphamide and fludarabine and then ARI-0001 cells, at first as a single dose and, after a protocol amendment, split into three fractions (10%, 30% and 60%) with the full dose given only in the absence of cytokine release syndrome.\n\nIn the 2021 report in Molecular Therapy, 58 patients were included and 47 treated: 38 with acute lymphoblastic leukaemia, 8 with non-Hodgkin lymphoma and 1 with chronic lymphocytic leukaemia. In acute lymphoblastic leukaemia, grade 3 or higher cytokine release syndrome occurred in 13.2% (26.7% before the amendment against 4.3% after), and the measurable residual disease-negative complete response rate at day 100 was 71.1%. The registry lists 50 participants and completion on 13 September 2022. On these results the AEMPS authorised ARI-0001 under the hospital exemption in February 2021 for adults over 25 with relapsed or refractory acute lymphoblastic leukaemia.","status":"completed","asOf":"2026-11-03","links":[{"label":"ClinicalTrials.gov NCT03144583","url":"https://clinicaltrials.gov/study/NCT03144583"},{"label":"Ortíz-Maldonado et al., Mol Ther 2021","url":"https://doi.org/10.1016/j.ymthe.2020.09.027"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/33010231/"}],"tags":[],"related":[],"cancers":["all-leukemia","dlbcl","cll"],"sections":[],"technologies":["car-t","point-of-care-cell-manufacturing"],"targets":["cd19"],"drugs":["varnimcabtagene-autoleucel"],"companies":[],"institutions":["hospital-clinic-barcelona","isciii"],"pathways":[],"terms":["crs","icans"],"trials":[],"people":["julio-delgado","valentin-ortiz-maldonado","manel-juan"],"bottlenecks":[],"keyPapers":["paper-cart19-be-01-mol-ther-2021"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT03144583","phase":"1","setting":"Pilot Study on the Infusion of ARI-0001 Cells in Patients With CD19+ Leukemia or Lymphoma Refractory to Therapy","sponsor":"Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), with the Instituto de Salud Carlos III","result":"Measurable residual disease-negative complete response at day 100 in 71.1% of patients with acute lymphoblastic leukaemia; one-year overall survival 68.6% (Mol Ther 2021).","started":"2017-06-15","startedType":"actual","yearReported":2020,"enrolled":50,"enrolledBasis":"registry","outcomes":[]},"route":"/trials/cart19-be-01/","neighbours":{"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"cll","kind":"cancer","name":"Chronic lymphocytic leukaemia","route":"/cancers/cll/"},{"id":"dlbcl","kind":"cancer","name":"Diffuse large B-cell lymphoma","route":"/cancers/dlbcl/"}],"technology":[{"id":"car-t","kind":"technology","name":"CAR-T cell therapy","route":"/technologies/car-t/"},{"id":"point-of-care-cell-manufacturing","kind":"technology","name":"Point-of-care and decentralised cell manufacturing","route":"/technologies/point-of-care-cell-manufacturing/"}],"target":[{"id":"cd19","kind":"target","name":"CD19","route":"/targets/cd19/"}],"drug":[{"id":"varnimcabtagene-autoleucel","kind":"drug","name":"Varnimcabtagene autoleucel","route":"/drugs/varnimcabtagene-autoleucel/"}],"institution":[{"id":"hospital-clinic-barcelona","kind":"institution","name":"Hospital Clínic de Barcelona / IDIBAPS","route":"/institutions/hospital-clinic-barcelona/"},{"id":"isciii","kind":"institution","name":"Instituto de Salud Carlos III (ISCIII)","route":"/institutions/isciii/"}],"term":[{"id":"crs","kind":"term","name":"Cytokine release syndrome (CRS)","route":"/terms/crs/"},{"id":"icans","kind":"term","name":"ICANS (neurotoxicity)","route":"/terms/icans/"}],"person":[{"id":"julio-delgado","kind":"person","name":"Julio Delgado","route":"/people/julio-delgado/"},{"id":"manel-juan","kind":"person","name":"Manel Juan","route":"/people/manel-juan/"},{"id":"valentin-ortiz-maldonado","kind":"person","name":"Valentín Ortiz-Maldonado","route":"/people/valentin-ortiz-maldonado/"}],"paper":[{"id":"paper-cart19-be-01-mol-ther-2021","kind":"paper","name":"CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19(+) Relapsed/Refractory Malignancies","route":"/key-papers/paper-cart19-be-01-mol-ther-2021/"},{"id":"paper-trias-hospital-exemption-ari-0001-bmt-2022","kind":"paper","name":"The hospital exemption pathway for advanced therapies and the case of the CAR-T ARI-0001","route":"/key-papers/paper-trias-hospital-exemption-ari-0001-bmt-2022/"}]}}