# AGILE

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

## TL;DR

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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-07
- Registry id: NCT03173248
- Phase: 3
- Setting: Newly diagnosed IDH1-mutated AML unfit for intensive chemotherapy: ivosidenib + azacitidine vs placebo + azacitidine
- Sponsor: Agios / Servier
- Enrolled: 146
- Result: OS 24.0 vs 7.9 months; HR 0.44.
- Outcomes: Event-free survival: Ivosidenib + azacitidine vs Placebo + azacitidine, HR 0.33; Overall survival (median): Ivosidenib + azacitidine 24 months vs Placebo + azacitidine 7.9 months, HR 0.44; Complete remission: Ivosidenib + azacitidine 47% vs Placebo + azacitidine 15%
- Replication: Single-arm ivosidenib-azacitidine (AG120-C-009) and venetoclax-azacitidine IDH subgroup data align; no second randomised trial yet.

## Sources

- ClinicalTrials.gov NCT03173248: https://clinicaltrials.gov/study/NCT03173248
- NEJM 2022: https://www.nejm.org/doi/full/10.1056/NEJMoa2117344

## Connected records

- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute myeloid leukaemia in older or unfit patients](https://onco.cc/cancers/aml-older-unfit/), [IDH1- and IDH2-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-idh/)
- technologies: [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/), [IDH inhibitors](https://onco.cc/technologies/idh-inhibitors/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [IDH1 / IDH2](https://onco.cc/targets/idh/)
- drugs: [Azacitidine](https://onco.cc/drugs/azacitidine/), [Ivosidenib](https://onco.cc/drugs/ivosidenib/)
- companies: [Servier](https://onco.cc/companies/servier/)
- people: [Courtney D. DiNardo](https://onco.cc/people/courtney-dinardo/)
- key papers: [AGILE: ivosidenib plus azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy](https://onco.cc/key-papers/paper-agile-ivosidenib-azacitidine-nejm-2022/)
- roadmaps: [Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome](https://onco.cc/roadmaps/epigenetics-roadmap/)

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