New drug combo targets tough leukemia cases

NCT ID NCT00819546

Ongoing Disease control Sponsor: Richard Stone, MD Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests the safety and best dose of combining two drugs—everolimus and midostaurin—for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or is hard to treat. The drugs work by blocking faulty signals that help cancer cells grow. The study includes 29 participants and aims to find the highest safe dose of everolimus when given with midostaurin.

What this could mean

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

Active substance: everolimus (RAD001) and midostaurin (PKC412)
What this could lead to: If this works, it could point toward a new treatment option for people with AML or MDS who have not responded to standard therapies.
What could go wrong: This is a very early phase 1 trial with only 29 participants, focused on safety and dosing. It is not designed to prove effectiveness, and the combination may cause side effects or fail to show 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 Myelodysplastic Syndromes

As listed by the trial registrant

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

ACUTE MYELOID LEUKEMIA MYELODYSPLASTIC SYNDROME

Contacts and locations

Locations

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

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