{"entity":{"id":"quazar-aml-001","kind":"trial","name":"QUAZAR AML-001","aka":["QUAZAR"],"tldr":"QUAZAR AML-001 showed that a tablet form of azacitidine taken as maintenance after chemotherapy helped older people with acute myeloid leukaemia live about ten months longer than placebo, and it became the first approved maintenance treatment for the disease.","summary":"QUAZAR AML-001 was a double-blind, placebo-controlled phase 3 trial in 472 patients aged 55 and over with acute myeloid leukaemia in first complete remission (with or without count recovery) after induction with or without consolidation, who were not candidates for stem-cell transplant. Patients were randomised to oral azacitidine 300 mg or placebo once daily for 14 days per 28-day cycle. The primary endpoint was overall survival.\n\nMedian overall survival was 24.7 months with oral azacitidine against 14.8 months with placebo, and relapse-free survival was also longer. Side effects were mainly gastrointestinal symptoms and neutropenia, and quality-of-life scores were maintained. Oral azacitidine (Onureg) was approved in the United States in September 2020 for continued treatment of adults with acute myeloid leukaemia in first remission who cannot complete intensive curative therapy.","status":"positive","asOf":"2026-09-22","links":[{"label":"ClinicalTrials.gov NCT01757535","url":"https://clinicaltrials.gov/study/NCT01757535"}],"tags":["soc-trials"],"related":[],"cancers":["aml","aml-older-unfit","leukaemia"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":["azacitidine"],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-quazar-aml-001-wei-nejm-2020"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT01757535","phase":"3","setting":"Acute myeloid leukaemia in first remission after intensive chemotherapy in patients aged 55 or older who were not going to transplant: oral azacitidine (CC-486) or placebo for 14 days of each 28-day cycle as maintenance","sponsor":"Celgene","result":"Median overall survival 24.7 months with oral azacitidine against 14.8 months with placebo (hazard ratio 0.69); approved in the United States in September 2020.","yearReported":2020,"enrolled":472,"enrolledBasis":"registry","outcomes":[{"endpoint":"Overall survival","primary":true,"unit":"months","arms":[{"name":"Oral azacitidine + best supportive care","n":238,"value":24.7,"note":"95% CI 18.7 to 30.5; median follow-up 41.2 months"},{"name":"Placebo + best supportive care","n":234,"value":14.8,"note":"95% CI 11.7 to 17.6"}],"hr":0.69,"ci":[0.55,0.86],"p":"0.0009","source":"https://doi.org/10.1056/nejmoa2004444"},{"endpoint":"Relapse-free survival (registry, August 2024 cut-off)","unit":"months","arms":[{"name":"Oral azacitidine + best supportive care","value":10.2},{"name":"Placebo + best supportive care","value":4.8}],"hr":0.65,"ci":[0.52,0.8],"p":"<0.0001","source":"https://clinicaltrials.gov/study/NCT01757535?tab=results"}]},"route":"/trials/quazar-aml-001/","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":"leukaemia","kind":"cancer","name":"Leukaemia (all types)","route":"/cancers/leukaemia/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"}],"drug":[{"id":"azacitidine","kind":"drug","name":"Azacitidine","route":"/drugs/azacitidine/"}],"company":[{"id":"bms","kind":"company","name":"Bristol Myers Squibb","route":"/companies/bms/"}],"paper":[{"id":"paper-quazar-aml-001-wei-nejm-2020","kind":"paper","name":"QUAZAR AML-001: oral azacitidine maintenance for acute myeloid leukaemia in first remission","route":"/key-papers/paper-quazar-aml-001-wei-nejm-2020/"}]}}