Targeted drug shows promise as maintenance after transplant for rare blood cancers

NCT ID NCT03515512

Completed Disease control Sponsor: Massachusetts General Hospital Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia chronic myelomonocytic leukemia myelodysplastic syndrome

As listed by the trial registrant

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

ACUTE MYELOID LEUKEMIA CHRONIC MYELOMONOCYTIC LEUKEMIA

Contacts and locations

Locations

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Johns Hopkins Cancer Center

Baltimore, Maryland, 21287, United States
Massachusetts General Hospital

Boston, Massachusetts, 02214, United States
Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

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