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Therapy-related Acute Myeloid Leukemia and Myelodysplastic SyndromePhase 25NCT02438761

PF-05212384 (PKI-587) for t-AML/MDS or de Novo Relapsed or Refractory Acute Myeloid Leukemia (AML) (LAM-PIK)

A Phase 2 study testing PF-05212384 in people with Therapy-related Acute Myeloid Leukemia and Myelodysplastic Syndrome and Acute Myeloid Leukemia, in Relapse, run by Institut Curie at 5 sites in France.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Phase II open-label single-arm prospective multicentric clinical trial of PF-05212384 (PKI-587) delivered by intravenous route. A 2-stage Fleming design will be employed. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
PF-05212384
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
5 sitesFrance
Study dates
31 Aug 2015to 23 Apr 2018
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
  • Adequate glycemic balance defined by glycated hemoglobin ≤ 8%
  • Females of childbearing potential (FCBP) should receive effective contraception: a negative pregnancy blood test is required within 2 weeks before starting experimental treatment.
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  • Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2
  • Absence of severe or active infection
  • Adequate systolic cardiac function : Left Ventricular Ejection Fraction (LVEF) ≥ 50%
  • 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
  • Adequate renal function: serum creatinine ≤ 1.5 x ULN or calculated creatinine clearance > 60 ml/min.
  • Signed informed consent

Who cannot join

  • Glucose intolerance or diabetes mellitus, treated or untreated
  • 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
  • AML secondary to MDS or myeloproliferative syndrome (WHO 2008 definitions)
  • Acute Promyelocytic Leukaemia (APL or AML French American British (FAB) classification 3) de novo or secondary to treatment (t-APL)
  • de novo or secondary Core Binding Factor (CBF)/AML
  • 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
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  • Leukocytes above 30.000/mm3 (30 G/L) at enrollment
  • Antileukemic treatment within 15 days before enrollment, with the exception of hydroxyurea
  • Central nervous system leukemic involvement
  • Pregnant or lactating women, or women of childbearing potential without effective contraception
  • Prior history of allogeneic bone marrow transplantation
  • Prior history of organ transplantation or other cause of severe or chronic immunodeficiency Human
  • Seropositivity for Human Immunodeficiency Virus (HIV) or Human T-Lymphotropic Virus-1 (HTLV-1) viruses, active B or C hepatitis
  • Inclusion in another experimental anti-cancer clinical trial*
  • Patients unable to undergo medical monitoring for geographical, social or psychological issues
  • Patient under measure of legal protection
  • 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.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

France5 sites

  • Institut Paoli Calmette

    Marseille, PACA, 13009

  • Hôpital Saint-Louis

    Paris, Île-de-France Region, 75475

  • Hôpital Cochin

    Paris, Île-de-France Region, 75679

  • Institut Curie - Hôpital René Huguenin

    Saint-Cloud, Île-de-France Region, 92210

  • CHU de Toulouse

    Toulouse, Midi-Pyrénées, 31059

Other trials in France

About this study

Official title: Phase II Evaluating the Efficacy of the Dual Inhibition of Phosphoinositide 3 Kinase (PI3K)/Akt /Mammalian Target Of Rapamycine (mTOR) Signaling Pathway by PF-05212384 (PKI-587) for Patients With Myeloid Neoplasm Secondary to Chemo-radiotherapy (t-AML/MDS) or de Novo Relapsed or Refractory AML.

Phase II open-label single-arm prospective multicentric clinical trial of PF-05212384 (PKI-587) delivered by intravenous route. A 2-stage Fleming design will be employed.

Read the detailed description

The treatment is administered in cycles of 28 days for a period of 4 cycles. Patients will be treated on a weekly basis continuously during 112 days or until progression.

Blood tests (hemogram) are assessed weekly before each injection of PF-05212384 (PKI-587). Bone marrow aspiration (myelogram) is performed to evaluate the response before starting treatment and before the start of cycle 3 (after two cycles) and at the end of the study (after four cycles). Good responders who continue treatment after four cycles will be evaluated by bone marrow aspiration (myelogram) every two cycles and after the end of treatment

Being tested
PF-05212384
Keywords
Acute Myeloid Leukemia, Myelodysplastic Syndrome, PIK/Akt/mTor
Medical subject headings
Myelodysplastic Syndromes, Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PF-05212384Drug

    Also called PKI-587

    PF-05212384 will be delivered by intra-venous route at a fixed dose of 150 mg per week. Each treatment cycle includes four weekly injections

    The treatment is administered in cycles of 28 days for a period of 4 cycles. Patients will be treated on a weekly basis continuously during 112 days or until progression.

Study groups in NCT02438761
Study groupRoleWhat they receive
PF-05212384Experimental150 mg Intra-venous every week
Receives: Drug: PF-05212384

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To evaluate the efficacy of PF-05212384

    The overall response rate will be assessed according to the International Working Group (IWG) AML and MDS criteria (by B.D. Cheson).

    Measured: 4 months after treatment

Secondary outcomes (5)

  1. Tolerance and toxicity during treatment

    Issued the Common Terminology Criteria for Adverse Events (CTCAE) version 4 National Cancer Institute (NCI)

    Measured: 4 months

  2. Treatment compliance

    Treatment compliance will be assessed by the ratio between the number of cycles administered on the expected number of cycles, and on time between treatment cycles

    Measured: 4 months

  3. Progressive Free Survival (PFS)

    Progressive Free Survival at one year from the date of inclusion to the date of progression of the disease or death

    Measured: one year

  4. Overall survival

    Overall Survival from the date of inclusion to the date of death

    Measured: 48 months

  5. Evaluation of Quality of life

    Quality of life (QLQ-C30) questionnaire according to European Organisation for Research and Treatment of Cancer (EORTC)

    Measured: 4 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 May 2015 (estimated)
  2. Study start31 Aug 2015
  3. Primary completion10 May 2017
  4. Study completion23 Apr 2018

Why it stopped: No objective response was observed at the first step. The treatment was considered ineffective, with a complete clinical trial suspension.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02438761
Sponsor's protocol ID
IC 2014-10
Acronym
5
Status last verified
Feb 2019
First submitted
6 May 2015
Last updated
8 Sep 2025
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PF-05212384 in people with Therapy-related Acute Myeloid Leukemia and Myelodysplastic Syndrome and Acute Myeloid Leukemia, in Relapse, run by Institut Curie at 5 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 8 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: No objective response was observed at the first step. The treatment was considered ineffective, with a complete clinical trial suspension.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To evaluate the efficacy of PF-05212384 (time frame: 4 months after treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02438761.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2025.

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