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

NCT ID NCT02438761

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

10 people

The number who actually took part.

Started

Aug 2015

Finished

Apr 2018

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Patients belong to one of three categories: * Myeloid neoplasm secondary to chemo-radiotherapy (t-AML/MDS) aged 60 and over with unfavorable cytogenetics (European Leukemia Network definition 2010), the first cancer must have been in remission for more than two years, except in situ carcinoma, basal cell carcinoma and squamous cell carcinoma * Relapsed or refractory de novo AML aged 18 and over (multiple relapses allowed), regardless of the risk group, provided not being eligible for allogeneic bone marrow transplantation * de novo AML at diagnosis, aged 60 and over and considered unfit to benefit from induction chemotherapy associated with aplasia (at the discretion of the investigator) 2. Adequate glycemic balance defined by glycated hemoglobin ≤ 8% 3. Females of childbearing potential (FCBP) should receive effective contraception: a negative pregnancy blood test is required within 2 weeks before starting experimental treatment. 4. Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2 5. Absence of severe or active infection 6. Adequate systolic cardiac function : Left Ventricular Ejection Fraction (LVEF) ≥ 50% 7. Adequate hepatic function: Aspartate Aminotransferase Test (AST) and Alanine Aminotransferase Test (ALT) ≤ 3 times the upper limit of normal (ULN), bilirubin ≤ 1.5 x ULN 8. Adequate renal function: serum creatinine ≤ 1.5 x ULN or calculated creatinine clearance \> 60 ml/min. 9. Signed informed consent Exclusion Criteria: 1. Glucose intolerance or diabetes mellitus, treated or untreated 2. First cancer in evolution(solid tumor or lymphoma) or in remission for less than two years, except in situ carcinoma, basal cell carcinoma and squamous cell carcinoma 3. AML secondary to MDS or myeloproliferative syndrome (WHO 2008 definitions) 4. Acute Promyelocytic Leukaemia (APL or AML French American British (FAB) classification 3) de novo or secondary to treatment (t-APL) 5. de novo or secondary Core Binding Factor (CBF)/AML 6. de novo or secondary Philadelphia Chromosome (Ph) 1 positive AML defined by the presence of a t(9.22) or a Breakpoint Cluster Region-Abelson Murine Leukemia Viral Oncogene Homolog (BCR-ABL) transcript 7. Leukocytes above 30.000/mm3 (30 G/L) at enrollment 8. Antileukemic treatment within 15 days before enrollment, with the exception of hydroxyurea 9. Central nervous system leukemic involvement 10. Pregnant or lactating women, or women of childbearing potential without effective contraception 11. Prior history of allogeneic bone marrow transplantation 12. Prior history of organ transplantation or other cause of severe or chronic immunodeficiency Human 13. Seropositivity for Human Immunodeficiency Virus (HIV) or Human T-Lymphotropic Virus-1 (HTLV-1) viruses, active B or C hepatitis 14. Inclusion in another experimental anti-cancer clinical trial\* 15. Patients unable to undergo medical monitoring for geographical, social or psychological issues 16. Patient under measure of legal protection 17. No social security * For ethical reasons, the exclusion period before considering the possibility of participating in another clinical study with a new experimental molecule cannot be determined, yet each case will be discussed on an individual basis with the study coordinator.

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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

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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