# AZA-001

Source: https://onco.cc/trials/aza-001/  
OnCo record `aza-001` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-17
- Tags: subtype-trials
- Registry id: NCT00071799
- Phase: 3
- Setting: Higher-risk myelodysplastic syndromes: azacitidine against a conventional care regimen (best supportive care, low-dose cytarabine or intensive chemotherapy) chosen for each patient before randomisation
- Sponsor: Celgene
- Enrolled: 358
- Result: Azacitidine improved overall survival over conventional care regimens in higher-risk myelodysplastic syndromes.

## Sources

- ClinicalTrials.gov NCT00071799: https://clinicaltrials.gov/study/NCT00071799

## Connected records

- cancers: [Higher-risk myelodysplastic syndromes](https://onco.cc/cancers/mds-higher-risk/), [Myelodysplastic syndromes / neoplasms (MDS)](https://onco.cc/cancers/mds/)
- drugs: [Azacitidine](https://onco.cc/drugs/azacitidine/)
- companies: [Bristol Myers Squibb](https://onco.cc/companies/bms/)
- people: [Pierre Fenaux](https://onco.cc/people/pierre-fenaux/)
- key papers: [AZA-001: azacitidine versus conventional care in higher-risk myelodysplastic syndromes](https://onco.cc/key-papers/paper-aza-001-fenaux-lancet-oncol-2009/)

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