Can a pill keep blood cancer at bay after a stem cell transplant?

NCT ID NCT04173533

Completed Disease control Sponsor: University of Birmingham Source: ClinicalTrials.gov ↗

First seen Aug 14, 2026 · Last updated Aug 14, 2026

Summary

This phase III trial is testing whether taking oral azacitidine as a maintenance therapy after an allogeneic stem cell transplant can help prevent relapse in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Participants are randomly assigned to receive either the drug or a placebo. The study aims to see if the drug improves relapse-free survival at one year and overall survival at one and two years, compared to placebo.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: oral azacitidine (CC-486)
What this could lead to: If effective, this could offer a way to keep leukemia and related blood cancers in remission longer after a stem cell transplant, potentially improving survival.
What could go wrong: This is an early-stage test in a specific post-transplant setting, and results may not apply to all patients. Side effects from the drug could also limit its use.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia chronic myelomonocytic leukemia myelodysplastic syndrome myelodysplastic syndrome with excess blasts therapy related acute myeloid leukemia and myelodysplastic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACUTE MYELOID LEUKEMIA MYELODYSPLASIA

Contacts and locations

Locations

Addenbrooke's Hospital

Cambridge, United Kingdom
Churchill Hospital

Oxford, United Kingdom
Clatterbridge Cancer Centre

Liverpool, United Kingdom
Derriford Hospital

Plymouth, United Kingdom
Freeman Hospital

Newcastle, United Kingdom
Hammersmith Hospital

London, United Kingdom
King's College Hospital

London, United Kingdom
Leicester Royal Infirmary

Leicester, United Kingdom
Manchester Royal Infirmary

Manchester, United Kingdom
Nottingham City Hospital

Nottingham, United Kingdom
Queen Elizabeth University Hospital

Glasgow, United Kingdom
Royal Hallamshire Hospital

Sheffield, United Kingdom
St Bartholomew's Hospital

London, United Kingdom
St. James's University Hospital

Leeds, United Kingdom
The Christie Hospital

Manchester, United Kingdom
The Queen Elizabeth Hospital

Birmingham, United Kingdom
The Royal Marsden Hospital

London, United Kingdom
University College London Hospitals

London, United Kingdom
University Hospital Bristol

Bristol, United Kingdom
University Hospital of Wales

Cardiff, United Kingdom

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