Experimental drug tested for Tough-to-Treat leukemia, but study stopped early
NCT ID NCT02438761
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 Hide 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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Acute myeloid leukemia, in relapse are added.
Genom att skicka in godkänner du våra Användarvillkor
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Blood cancer registry tracks thousands to map disease course
- Can a Two-Drug attack outsmart resistant leukemia?
- Engineered immune cells take aim at Hard-to-Treat leukemia
- Can a simple blood test predict leukemia risk after cancer therapy?
- Could a pill keep AML at bay? new study tests maintenance therapy after chemo
- Promising drug combo targets tough leukemia in early trial