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

• ClinicalTrials.gov Identifier: NCT01174043
• Recruitment Status: Completed
• First Posted: August 3, 2010
• Results First Posted: March 27, 2015
• Last Update Posted: March 27, 2015
• Sponsor: Indiana University
• Collaborator: OSI Pharmaceuticals
• Information provided by (Responsible Party): Indiana University

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Leukemia, Myelomonocytic, Acute	Drug: Erlotinib	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Pilot Study of Erlotinib for the Treatment of Patients With de Novo Acute Myeloid Leukemia
• Study Start Date: July 2010
• Actual Primary Completion Date: March 2012
• Actual Study Completion Date: March 2012

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures :

1. Overall Response Rate (Defined as Partial Remission or Better) to 3 Months of Treatment With Erlotinib [ Time Frame: 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.

Secondary Outcome Measures :

1. Duration of Response (up to One Year Follow up) in Patients Who Achieve a Complete Remission [ Time Frame: 1 year after treatment discontinuation ]
   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.
2. Treatment Related Adverse Events Grade 3 or Higher [ Time Frame: up to 15 months ]
   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.

Other Outcome Measures:

1. 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 [ Time Frame: Baseline; days 3, 4, 8, and 29 of course 1; and day 29 of courses 3, 6, 9, and 12 ]

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

United States, Indiana

Indiana University Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, United States, 46202

Sponsors and Collaborators

Indiana University

OSI Pharmaceuticals

Investigators

• Principal Investigator: S. Hamid Sayar, MD; Indiana University Melvin and Bren Simon Cancer Center

More Information

• Responsible Party: Indiana University
• ClinicalTrials.gov Identifier: NCT01174043
• Other Study ID Numbers: 1006-12; IUCRO-0300
• First Posted: August 3, 2010
• Results First Posted: March 27, 2015
• Last Update Posted: March 27, 2015
• Last Verified: March 2015

Keywords provided by Indiana University:

Leukemia, Myelomonocytic, Acute; Erlotinib

Additional relevant MeSH terms:

Leukemia; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Acute; Neoplasms by Histologic Type; Neoplasms; Leukemia, Myeloid; Erlotinib Hydrochloride; Antineoplastic Agents; Protein Kinase Inhibitors; Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action