{"entity":{"id":"agile","kind":"trial","name":"AGILE","aka":[],"tldr":"In IDH1-mutated AML, adding ivosidenib to azacitidine tripled survival, one of the largest effects ever seen in a randomised AML trial.","summary":"Event-free survival HR 0.33 (95% CI 0.16-0.69); median OS 24.0 vs 7.9 months (HR 0.44, 95% CI 0.27-0.73); CR 47% vs 15%. Stopped early for benefit. NEJM 2022. Whether ivosidenib-azacitidine or venetoclax-azacitidine (which also works well in IDH-mutant AML) should be first line remains debated; triplets are being tested.","status":"positive","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT03173248","url":"https://clinicaltrials.gov/study/NCT03173248"},{"label":"NEJM 2022","url":"https://www.nejm.org/doi/full/10.1056/NEJMoa2117344"}],"tags":[],"related":[],"cancers":["aml","aml-idh"],"sections":[],"technologies":["epigenetic-drugs","idh-inhibitors","kinase-inhibitors"],"targets":["idh"],"drugs":["ivosidenib","azacitidine"],"companies":["servier"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":["courtney-dinardo"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT03173248","phase":"3","setting":"Newly diagnosed IDH1-mutated AML unfit for intensive chemotherapy: ivosidenib + azacitidine vs placebo + azacitidine","sponsor":"Agios / Servier","result":"OS 24.0 vs 7.9 months; HR 0.44.","started":"2017-06-26","startedType":"actual","yearReported":2022,"enrolled":146,"enrolledBasis":"registry","outcomes":[{"endpoint":"Event-free survival","primary":true,"arms":[{"name":"Ivosidenib + azacitidine","n":72},{"name":"Placebo + azacitidine","n":74}],"hr":0.33,"ci":[0.16,0.69],"p":"0.002","source":"https://www.nejm.org/doi/full/10.1056/NEJMoa2117344"},{"endpoint":"Overall survival (median)","unit":"months","arms":[{"name":"Ivosidenib + azacitidine","value":24},{"name":"Placebo + azacitidine","value":7.9}],"hr":0.44,"ci":[0.27,0.73],"source":"https://doi.org/10.1056/NEJMoa2117344"},{"endpoint":"Complete remission","unit":"%","arms":[{"name":"Ivosidenib + azacitidine","value":47},{"name":"Placebo + azacitidine","value":15}],"source":"https://doi.org/10.1056/NEJMoa2117344"}],"replication":"Single-arm ivosidenib-azacitidine (AG120-C-009) and venetoclax-azacitidine IDH subgroup data align; no second randomised trial yet."},"route":"/trials/agile/","neighbours":{"cancer":[{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"aml-older-unfit","kind":"cancer","name":"Acute myeloid leukaemia in older or unfit patients","route":"/cancers/aml-older-unfit/"},{"id":"aml-idh","kind":"cancer","name":"IDH1- and IDH2-mutated acute myeloid leukaemia","route":"/cancers/aml-idh/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"},{"id":"idh-inhibitors","kind":"technology","name":"IDH inhibitors","route":"/technologies/idh-inhibitors/"},{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"idh","kind":"target","name":"IDH1 / IDH2","route":"/targets/idh/"}],"drug":[{"id":"azacitidine","kind":"drug","name":"Azacitidine","route":"/drugs/azacitidine/"},{"id":"ivosidenib","kind":"drug","name":"Ivosidenib","route":"/drugs/ivosidenib/"}],"company":[{"id":"servier","kind":"company","name":"Servier","route":"/companies/servier/"}],"person":[{"id":"courtney-dinardo","kind":"person","name":"Courtney D. DiNardo","route":"/people/courtney-dinardo/"}],"paper":[{"id":"paper-agile-ivosidenib-azacitidine-nejm-2022","kind":"paper","name":"AGILE: ivosidenib plus azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy","route":"/key-papers/paper-agile-ivosidenib-azacitidine-nejm-2022/"}],"roadmap":[{"id":"epigenetics-roadmap","kind":"roadmap","name":"Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome","route":"/roadmaps/epigenetics-roadmap/"}]}}