Pilot Study of Erlotinib for the Treatment of Patients With de Novo Acute Myeloid Leukemia

This research study is looking for patients with newly diagnosed acute myeloid leukemia (AML), AML that has returned (relapsed), or it has not responded adequately to previous treatments. Treating certain patients with chemotherapy may not be to their benefit or may cause more harm than benefit. The purpose of this study is to find out what effects (good and bad) erlotinib has on patients and their AML.

Study Overview

• Status: Completed
• Conditions: Leukemia, Myelomonocytic, Acute
• Intervention / Treatment: Drug: Erlotinib

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Melvin and Bren Simon Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of AML with no history of previous clonal/malignant hematologic disorders such as myelodysplastic syndromes or myeloproliferative disorders.
• Newly diagnosed patients will be age 70 or older
• Relapses patients will be age 60 or older any time following first relapse, if patient is not considered candidate/not interested in salvage chemotherapy.
• Refractory disease patients will be age 18-59 who have failed at least 2 lines of conventional chemotherapy (1 induction and 1 salvage)
• Patient must have discontinued all previous therapies for AML at least 14 days and recovered from the non-hematologic side effects of the therapy.
• Laboratory tests must be within protocol-specified ranges
• Patient must be able to swallow and tolerate oral medication.

Exclusion Criteria:

• Patients with known central nervous system (CNS) leukemia by spinal fluid cytology, flow cytometry or imaging.
• History of antecedent pre-leukemic hematologic disorders such as myelodysplastic syndromes or myeloproliferative disorders.
• Diagnosis of acute promyelocytic leukemia (APL)
• Patients who require chronic anticoagulation, are current smokers or who are taking rifabutin, rifapentine, phenytoin, carbamazepine, phenobarbital and St. John's Wort are not eligible.
• Patients with active corneal erosions or history of abnormal corneal sensitivity test.
• Patients with serious illness such as: significant ongoing or active infection, New York Heart Association (NYHA) Grade II or greater congestive heart failure, unstable angina (anginal symptoms at rest), new onset angina (began within the last 3 months), myocardial infarction within the past 6 months, cardiac ventricular arrhythmias requiring anti-arrhythmic therapy, cerebrovascular accident within past 3 months, or psychiatric illness that would limit compliance with the study requirements.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Erlotinib	Drug: Erlotinib; Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate (Defined as Partial Remission or Better) to 3 Months of Treatment With Erlotinib	The percent of patients were shown as having a partial remission or better based on definitions of response in AML. Partial remission includes a decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate. Complete remission includes presence of less than 5% blasts in an aspirate sample with marrow spicules and with a count of at least 200 nucleated cells. The percent and 95% exact confidence intervals will be calculated.	3 months of treatment with erlotinib

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Duration of Response (up to One Year Follow up) in Patients Who Achieve a Complete Remission	The duration of response is from the time of response until failure or until the end of follow-up for the patients who received complete remission. Complete remission includes presence of less than 5% blasts in an aspirate sample with marrow spicules and with a count of at least 200 nucleated cells.	1 year after treatment discontinuation
Treatment Related Adverse Events Grade 3 or Higher	Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. Grading scale will be from 1 (mild) to 5 (causing death). This will determine the number of unique patients who had a treatment related (possible, probable or definite) adverse event that was graded 3 or greater.	up to 15 months

Other Outcome Measures

Outcome Measure	Time Frame
Mechanistic Attributes of Erlotinib Hydrochloride in AML, Including Intracellular Quantitative Protein and Gene Expression Modifications and the in Vivo Effect of This Agent on the Differentiation of AML Blasts	Baseline; days 3, 4, 8, and 29 of course 1; and day 29 of courses 3, 6, 9, and 12

Collaborators and Investigators

• Sponsor: Indiana University
• Collaborators: OSI Pharmaceuticals
• Investigators: Principal Investigator: S. Hamid Sayar, MD, Indiana University Melvin and Bren Simon Cancer Center

Study record dates

Study Major Dates

• Study Start: July 1, 2010
• Primary Completion (Actual): March 1, 2012
• Study Completion (Actual): March 1, 2012

Study Registration Dates

• First Submitted: July 30, 2010
• First Submitted That Met QC Criteria: July 30, 2010
• First Posted (Estimated): August 3, 2010

Study Record Updates

• Last Update Posted (Actual): October 13, 2023
• Last Update Submitted That Met QC Criteria: October 11, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Erlotinib; Leukemia, Myelomonocytic, Acute
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia, Myeloid; Leukemia; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Acute; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Erlotinib Hydrochloride
• Other Study ID Numbers: 1006-12; IUCRO-0300