Experimental drug tested for Tough-to-Treat leukemia, but study stopped early

NCT ID NCT02438761

Terminated Disease control Sponsor: Institut Curie Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This phase 2 trial tested a drug called PF-05212384 in 10 people with certain types of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), including cases linked to past cancer treatment or that had come back after other therapies. The drug was given weekly through an IV for up to 4 months. The study was terminated early, so we have limited information on how well it worked or its side effects.

What this could mean

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

Active substance: PF-05212384 (PKI-587)
What this could lead to: If successful, this drug could offer a new treatment option for patients with hard-to-treat AML or MDS, especially those who have relapsed or not responded to other therapies.
What could go wrong: This was a very small, early-phase trial that was terminated before completion, so results are limited. The drug may cause significant side effects, and its effectiveness is not yet proven.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia Myelodysplastic Syndromes therapy related acute myeloid leukemia and myelodysplastic syndrome therapy-related myeloid neoplasm

As listed by the trial registrant

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

ACUTE MYELOID LEUKEMIA, IN RELAPSE DE NOVO ACUTE MYELOID LEUKEMIA AT DIAGNOSTIC THERAPY-RELATED ACUTE MYELOID LEUKEMIA AND MYELODYSPLASTIC SYNDROME

Contacts and locations

Locations

CHU de Toulouse

Toulouse, Midi-Pyrénées, 31059, France
Hôpital Cochin

Paris, Île-de-France Region, 75679, France
Hôpital Saint-Louis

Paris, Île-de-France Region, 75475, France
Institut Curie - Hôpital René Huguenin

Saint-Cloud, Île-de-France Region, 92210, France
Institut Paoli Calmette

Marseille, PACA, 13009, France

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