New targeted therapy shows promise for Tough-to-Treat leukemia

NCT ID NCT02204085

Completed Disease control Sponsor: Beth Israel Deaconess Medical Center Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called GO-203-2C, which targets a protein (MUC1) found on cancer cells while sparing healthy cells. It was given alone or with another drug (decitabine) to 33 adults with acute myeloid leukemia that had returned or not responded to treatment. The main goals were to find the safest dose and check for early signs of effectiveness.

What this could mean

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

Active substance: GO-203-2C (a targeted drug that blocks MUC1 protein in cancer cells), given alone or with decitabine
What this could lead to: If successful, this could point toward a new treatment option for patients with hard-to-treat acute myeloid leukemia who have not responded to standard therapy.
What could go wrong: This is an early-phase trial with only 33 participants, focused on safety and dosing. The drug may not prove effective enough, and side effects or lack of response are possible.

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

As listed by the trial registrant

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

ACUTE MYELOID LEUKEMIA, IN RELAPSE RECURRENT ADULT ACUTE MYELOID LEUKEMIA

Contacts and locations

Locations

Beth Israel Deaconess Medical Center

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

Boston, Massachusetts, 02115, United States

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