Drug duo shows promise for Tough-to-Treat leukemia

NCT ID NCT01869114

Completed Disease control Sponsor: Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This phase II trial tested two drugs—sirolimus and azacitidine—in 57 people with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that had come back or didn't respond to standard treatment. The goal was to see if the combination could shrink or control the cancer. The study measured how many patients had a meaningful response, such as improved blood counts or remission. It also tracked side effects to understand the safety of this approach.

What this could mean

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

Active substance: sirolimus and azacitidine
What this could lead to: If it works, this combination could offer a new treatment option for people with high-risk MDS or relapsed AML who cannot tolerate intensive chemotherapy.
What could go wrong: This is a small, completed phase II trial, so results are preliminary. The combination may not work for everyone and can cause side effects like low blood counts or infections.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia acute myeloid leukemia with 11q23 abnormalities acute myeloid leukemia with t(8;21)(q22;q22) translocation acute myeloid leukemia, del(5q31-q32) acute myeloid leukemia, inv(16)(p13.1;q22) acute myeloid leukemia, t(16;16)(p13.1;q22) acute promyelocytic leukemia Congenital Abnormalities myelodysplastic syndrome myelodysplastic syndrome associated with isolated del(5q)

As listed by the trial registrant

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

ADULT ACUTE MYELOID LEUKEMIA WITH 11Q23 (MLL) ABNORMALITIES ADULT ACUTE MYELOID LEUKEMIA WITH DEL(5Q) ADULT ACUTE MYELOID LEUKEMIA WITH INV(16)(P13;Q22) ADULT ACUTE MYELOID LEUKEMIA WITH T(15;17)(Q22;Q12) ADULT ACUTE MYELOID LEUKEMIA WITH T(16;16)(P13;Q22) ADULT ACUTE MYELOID LEUKEMIA WITH T(8;21)(Q22;Q22) DE NOVO MYELODYSPLASTIC SYNDROMES MYELODYSPLASTIC SYNDROME WITH ISOLATED DEL(5Q) PREVIOUSLY TREATED MYELODYSPLASTIC SYNDROMES RECURRENT ADULT ACUTE MYELOID LEUKEMIA

Contacts and locations

Locations

Abington Hospital - Jefferson Health

Abington, Pennsylvania, 19001, United States
Jefferson Health NJ Division (Kennedy Hospital)

Sewell, New Jersey, 08012, United States
Jefferson Health, Aria Hospital

Philadelphia, Pennsylvania, 19124, United States
Jefferson Health, Methodist Hospital

Philadelphia, Pennsylvania, 19148, United States
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

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