New drug combo targets tough leukemia cases
NCT ID NCT00819546
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.
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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Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02115, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States