Targeted drug shows promise as maintenance after transplant for rare blood cancers
NCT ID NCT03515512
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 1 study tested the drug enasidenib as a maintenance therapy after a stem cell transplant in 23 people with IDH2-mutant acute myeloid leukemia or chronic myelomonocytic leukemia. The goal was to find the safest dose and see how well patients tolerated it. Enasidenib works by blocking a mutated protein that can fuel cancer growth. The study was completed, and results help guide future research on using targeted therapy to prevent relapse after transplant.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Enasidenib (a targeted therapy drug that blocks the mutated IDH2 protein, which can fuel cancer growth)
What this could lead to
If this works, it could offer a way to keep IDH2-mutant blood cancers in remission after a stem cell transplant, potentially reducing the chance of relapse.
What could go wrong
This is a very early (phase 1) study with only 23 participants, so we don't yet know if enasidenib truly prevents relapse. The drug may cause side effects, and not all patients may benefit.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Johns Hopkins Cancer Center
Baltimore, Maryland, 21287, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02214, United States
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Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States