MEK Inhibitor MEK162, Idarubicin, and Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

NCT ID: NCT02049801

Last Updated: 2017-12-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-12-31

Study Completion Date

2017-11-01

Brief Summary

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This phase I trial studies the MEK inhibitor MEK162 to see if it is safe in patients when combined with idarubicin and cytarabine. MEK inhibitor MEK162 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as idarubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving MEK inhibitor MEK162, cytarabine, and idarubicin may be an effective treatment for acute myeloid leukemia.

Detailed Description

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PRIMARY OBJECTIVES:

I. Determine maximum tolerated dose (MTD) of MEK162 (MEK inhibitor MEK162) in patients with RAS-mutated acute myeloid leukemia (AML) when combined with sequential induction chemotherapy (3+4) as measured by development of grade 3-4 dose-limiting toxicities (DLT).

SECONDARY OBJECTIVES:

I. Analyze downstream inhibition of RAS signaling following therapy with single-agent MEK162 with exploratory pharmacodynamics (PD) studies.

II. Perform preliminary efficacy analysis of combination of MEK162 and induction chemotherapy (3+4) in patients with RAS-mutated AML by measuring complete remission rate, 2-year overall survival, and duration of response.

OUTLINE:

INDUCTION THERAPY: Patients receive MEK inhibitor MEK162 orally (PO) twice daily (BID) on days -4 to -1 and days 5-18, cytarabine intravenously (IV) continuously over 24 hours on days 1-4, and idarubicin IV over 1 hour on days 1-3. Patients may receive a second course of induction at the discretion of the principal investigator.

POST-REMISSION THERAPY: Patients receive cytarabine IV continuously over 24 hours on days 1-3, idarubicin IV over 1 hour on days 1 and 2, and MEK inhibitor MEK 162 PO BID on days 4-17. Treatment repeats every 28 days for up to 4 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for 30 days.

Conditions

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Adult Acute Minimally Differentiated Myeloid Leukemia (M0) Adult Acute Monoblastic Leukemia (M5a) Adult Acute Monocytic Leukemia (M5b) Adult Acute Myeloblastic Leukemia With Maturation (M2) Adult Acute Myeloblastic Leukemia Without Maturation (M1) Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities Adult Acute Myeloid Leukemia With Del(5q) Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) Adult Acute Myelomonocytic Leukemia (M4) Adult Erythroleukemia (M6a) Adult Pure Erythroid Leukemia (M6b) Recurrent Adult Acute Myeloid Leukemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment (MEK inhibitor, MEK162, idarubicin, cytarabine)

INDUCTION THERAPY: Patients receive MEK inhibitor MEK162 PO BID on days -4 to -1 and days 5-18, cytarabine IV continuously over 24 hours on days 1-4, and idarubicin IV over 1 hour on days 1-3. Patients may receive a second course of induction at the discretion of the principal investigator.

POST-REMISSION THERAPY: Patients receive cytarabine IV continuously over 24 hours on days 1-3, idarubicin IV over 1 hour on days 1 and 2, and MEK inhibitor MEK 162 PO BID on days 4-17. Treatment repeats every 28 days for up to 4 courses in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

MEK inhibitor MEK162

Intervention Type DRUG

Given PO

idarubicin

Intervention Type DRUG

Given IV

cytarabine

Intervention Type DRUG

Given IV

pharmacological study

Intervention Type OTHER

Correlative studies

laboratory biomarker analysis

Intervention Type OTHER

Correlative studies

Interventions

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MEK inhibitor MEK162

Given PO

Intervention Type DRUG

idarubicin

Given IV

Intervention Type DRUG

cytarabine

Given IV

Intervention Type DRUG

pharmacological study

Correlative studies

Intervention Type OTHER

laboratory biomarker analysis

Correlative studies

Intervention Type OTHER

Other Intervention Names

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ARRY-162 ARRY-438162 4-demethoxydaunorubicin 4-DMDR DMDR IDA ARA-C arabinofuranosylcytosine arabinosylcytosine Cytosar-U cytosine arabinoside pharmacological studies

Eligibility Criteria

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Inclusion Criteria

* Prior morphological diagnosis of AML other than acute promyelocytic leukemia according to the 2001 World Health Organization (WHO) diagnostic criteria; patients with biphenotypic, RAS-mutated acute leukemia are also eligible
* Requiring salvage chemotherapy for persistent/refractory or relapsed disease after at least one course of conventional chemotherapy, e.g. with "7+3", as defined by persistence of \>= 20% myeloid blasts on bone marrow aspirate or peripheral blood smear; a bone marrow biopsy is not routinely required, but should be obtained if the aspirate is dilute, hypocellular, or inaspirable; outside bone marrow examinations performed within the stipulated time period are acceptable for screening as long as the slides are reviewed at the study institution; flow cytometric analysis of the bone marrow aspirate should be performed according to institutional practice guidelines
* Confirmed RAS mutation (NRAS or KRAS) or confirmed PTPN11 mutation, measured on peripheral blood or bone marrow aspirate as part of screening prior to study enrollment; mutation status must be confirmed within 45 days of initiation of therapy
* Eastern Cooperative Oncology Group (ECOG) performance status =\< 1
* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 2.5 times upper limit of normal (ULN)
* Serum bilirubin =\< 2 times ULN
* Serum creatinine =\< 1.5 mg/dl and/or creatinine clearance \>= 50 mL/min
* Ejection fraction \>= 50% by echocardiogram
* Corrected QT (QTc) interval =\< 480 ms
* Ability to take oral medications
* Ability to understand and the willingness to sign a written informed consent document
* Ability to undergo standard induction chemotherapy
* Ability to adhere to the study visit schedule and other protocol requirements
* Life expectancy of greater than 2 months
* Negative serum beta-human chorionic gonadotropin (HCG) test (female patient of childbearing potential only) performed locally within 72 hrs prior to first dose

Exclusion Criteria

* Concomitant treatment with other anti-neoplastic agents, with the exception of hydroxyurea
* Anti-neoplastic treatment less than 14 days prior to enrollment, with the exceptions of hydroxyurea or, in the case of a patient with primary refractory AML, prior induction chemotherapy
* Prior therapy with a MEK inhibitor
* Uncontrolled opportunistic infection or treatment for opportunistic infection within 4 weeks of first day of study drug dosing
* Other medical or psychiatric illness or organ dysfunction or laboratory abnormality which in the opinion of the investigator would compromise the patient's safety or interfere with data interpretation, e.g., infection/inflammation, intestinal obstruction, unable to swallow medication, social/ psychological issues, etc.
* History of allergic reactions attributed to compounds of similar chemical or biologic composition to MEK162, anthracycline, or cytarabine
* Known impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of MEK162 (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection)
* Previous or concurrent malignancy with the following exceptions:

* Carcinoma in situ
* Adequately treated skin basal cell or squamous cell carcinoma (adequate wound healing is required prior to study entry)
* In situ carcinoma of the cervix, treated curatively and without evidence of recurrence for at least 3 years prior to the study
* A primary malignancy which has been completely resected and in complete remission for \>= 5 years
* Impaired cardiovascular function or clinically significant cardiovascular diseases, including any of the following:

* History of acute coronary syndromes (including myocardial infarction, unstable angina, coronary artery bypass grafting \[CABG\], coronary angioplasty, or stenting) \< 6 months prior to screening
* Symptomatic chronic heart failure
* Evidence of clinically significant cardiac arrhythmias and/or conduction abnormalities \< 6 months prior to screening except atrial fibrillation and paroxysmal supraventricular tachycardia
* Uncontrolled arterial hypertension despite appropriate medical therapy
* Patients who have neuromuscular disorders that are associated with elevated creatine kinase (CK) (e.g., inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis, spinal muscular atrophy)
* Known confirmed diagnosis of human immunodeficiency virus (HIV) infection or active viral hepatitis (hepatitis B or hepatitis C)
* Any surgical procedure, excluding central venous catheter placement, bone marrow biopsy, lumbar puncture, or other minor procedures (e.g., skin biopsy) within 14 days of day 1; patients who have undergone major surgery =\< 21 days prior to starting study drug or who have not recovered from side effects of such procedure are ineligible for the study
* Pregnant or nursing (lactating) women confirmed by a positive hCG laboratory test

* Women of child-bearing potential unless they are using highly effective methods of contraception throughout the study and for 30 days after study drug discontinuation
* Sexually active males unless they use a condom during intercourse while taking the drug and for 30 days after stopping treatment and should not father a child in this period; a condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid
* Unwillingness or inability to comply with the protocol
* Known active leukemia of the central nervous system
* Known history of Gilbert's syndrome
* History or current evidence of retinal pigment epithelial detachment (RPED), retinal vein occlusion (RVO), or predisposing factors to RPED or RVO (e.g. uncontrolled glaucoma or ocular hypertension, uncontrolled diabetes mellitus, hyperviscosity or hypercoagulability syndromes)
* History of retinal degenerative disease
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Bruno C. Medeiros

OTHER

Sponsor Role lead

Responsible Party

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Bruno C. Medeiros

Assistant Professor of Medicine

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Bruno de Medeiros

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

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Stanford University, School of Medicine

Stanford, California, United States

Site Status

Countries

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United States

Other Identifiers

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NCI-2014-00169

Identifier Type: REGISTRY

Identifier Source: secondary_id

HEMAML0030

Identifier Type: OTHER

Identifier Source: secondary_id

IRB-29150

Identifier Type: -

Identifier Source: org_study_id