{"entity":{"id":"eliana","kind":"trial","name":"ELIANA","aka":[],"tldr":"ELIANA was the global trial behind the first CAR-T approval: a single infusion of tisagenlecleucel put four in five children and young adults with relapsed B-cell acute lymphoblastic leukaemia and no standard treatment remaining into remission, and most responders were still in remission without further therapy at the five-year update. Severe cytokine release syndrome affected almost half.","summary":"ELIANA, trial NCT02435849 sponsored by Novartis and published in the New England Journal of Medicine in 2018, was the global trial behind the first CAR-T approval: four in five children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia and no standard treatment remaining went into remission after a single tisagenlecleucel infusion, and most responders stayed there for years. 75 patients were infused for the primary analysis (79 by the three-year update), the remission rate within three months was 81 percent with all responders MRD-negative, and the five-year update in JCO in 2023 showed durable survival in most responders without further therapy, at the cost of severe cytokine release syndrome in almost half. Whether children who relapse can be rescued with a second CAR-T is the open question.","status":"positive","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT02435849","url":"https://clinicaltrials.gov/study/NCT02435849"}],"tags":[],"related":[],"cancers":["all-leukemia","all-paediatric-relapsed"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":["tisagenlecleucel"],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["crs"],"trials":[],"people":["stephan-grupp","emily-whitehead"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT02435849","phase":"2","setting":"Relapsed or refractory B-ALL, patients aged 3-25: single infusion of tisagenlecleucel","sponsor":"Novartis","result":"ORR 81%; 12-month OS 76%; 5-year OS 55%.","started":"2015-04-08","startedType":"actual","yearReported":2018,"enrolled":79,"enrolledBasis":"treated","enrolledNote":"ClinicalTrials.gov lists 80 participants (actual); the JCO 2023 three-year update reports 79 infused patients and the NEJM 2018 primary analysis 75.","outcomes":[{"endpoint":"Overall remission rate within 3 months","primary":true,"unit":"%","arms":[{"name":"Tisagenlecleucel","n":75,"value":81}],"source":"https://www.nejm.org/doi/full/10.1056/NEJMoa1709866"},{"endpoint":"Overall survival at 12 months","unit":"%","arms":[{"name":"Tisagenlecleucel","value":76}],"source":"https://doi.org/10.1056/NEJMoa1709866"},{"endpoint":"Overall survival at 5 years","unit":"%","arms":[{"name":"Tisagenlecleucel","value":55}],"source":"https://ascopubs.org/doi/10.1200/JCO.22.00642"}],"replication":"ENSIGN (US) and real-world CIBMTR registry data (>500 children) reproduce ~85% CR and comparable durability."},"route":"/trials/eliana/","neighbours":{"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"all-paediatric-relapsed","kind":"cancer","name":"Relapsed and refractory acute lymphoblastic leukaemia in children","route":"/cancers/all-paediatric-relapsed/"}],"technology":[{"id":"car-t","kind":"technology","name":"CAR-T cell therapy","route":"/technologies/car-t/"}],"target":[{"id":"cd19","kind":"target","name":"CD19","route":"/targets/cd19/"}],"drug":[{"id":"tisagenlecleucel","kind":"drug","name":"Tisagenlecleucel","route":"/drugs/tisagenlecleucel/"}],"company":[{"id":"novartis","kind":"company","name":"Novartis","route":"/companies/novartis/"}],"term":[{"id":"crs","kind":"term","name":"Cytokine release syndrome (CRS)","route":"/terms/crs/"}],"person":[{"id":"emily-whitehead","kind":"person","name":"Emily Whitehead","route":"/people/emily-whitehead/"},{"id":"stephan-grupp","kind":"person","name":"Stephan A. Grupp","route":"/people/stephan-grupp/"}],"bottleneck":[{"id":"b-manufacturing-cell-therapy","kind":"bottleneck","name":"Manufacturing cost and time for living and radioactive medicines","route":"/bottlenecks/b-manufacturing-cell-therapy/"}],"paper":[{"id":"paper-eliana-tisagenlecleucel-nejm-2018","kind":"paper","name":"ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL","route":"/key-papers/paper-eliana-tisagenlecleucel-nejm-2018/"}],"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}]}}