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AZA-001 was the trial that showed a drug could help people with higher-risk myelodysplastic syndromes live longer: azacitidine beat the best conventional care, and it has been the backbone of treatment for this disease ever since.
AZA-001 was an open-label phase 3 trial in patients with higher-risk myelodysplastic syndromes (intermediate-2 or high risk by the International Prognostic Scoring System). Before randomisation the investigator chose the conventional care regimen the patient would otherwise receive: best supportive care, low-dose cytarabine or standard intensive chemotherapy. Patients were then randomised to subcutaneous azacitidine or to that regimen.
Azacitidine prolonged overall survival compared with conventional care, the primary endpoint, and delayed transformation to acute myeloid leukaemia. It was the first treatment to show a survival benefit in higher-risk MDS and the reason hypomethylating agents became the standard against which venetoclax combinations and newer agents are now tested.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
The full inclusion and exclusion criteria are on the registry record. Read 2026-10-07. Recruiting overall does not mean a particular site is open; ask the study team.
ClinicalTrials.gov data courtesy of the US National Library of Medicine.
Shares Pierre Fenaux, Myelodysplastic syndromes / neoplasms (MDS), Bristol Myers Squibb and the tag subtype-trials.
Shares Higher-risk myelodysplastic syndromes, Azacitidine, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Shares Myelodysplastic syndromes / neoplasms (MDS), Bristol Myers Squibb and the tag subtype-trials.
Shares Bristol Myers Squibb and the tag subtype-trials.
Shares Bristol Myers Squibb and the tag subtype-trials.
Shares Bristol Myers Squibb and the tag subtype-trials.
Shares Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET) and the tag subtype-trials.
Every source this page cites, grouped by who published it. Grouping is read from the web address, not assigned by hand.
A source that has moved or gone is reported by the weekly link check. /audit/