New drug combo targets tough leukemia cases in early trial

NCT ID NCT00819546

First seen Apr 29, 2026 · Last updated May 02, 2026 · Updated 1 time

Summary

This early-phase study tests the safety of combining two drugs, RAD001 and PKC412, in about 29 adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has returned or not responded to standard treatments. The goal is to find the highest safe dose of RAD001 when given with PKC412, which blocks a faulty enzyme (FLT3) that helps cancer cells grow, while RAD001 targets another growth pathway (mTOR). This is not a cure, but aims to control the disease by slowing cancer cell growth.

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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

Conditions

Explore the condition pages connected to this study.