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Erlotinib Hydrochloride in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01664897
Recruitment Status : Completed
First Posted : August 14, 2012
Results First Posted : January 7, 2020
Last Update Posted : January 7, 2020
National Cancer Institute (NCI)
Astellas Pharma Inc
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
This pilot phase II trial studies how well erlotinib hydrochloride works in treating patients with relapsed or refractory acute myeloid leukemia. Erlotinib hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 Adult Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A Adult Acute Promyelocytic Leukemia With PML-RARA Alkylating Agent-Related Acute Myeloid Leukemia Chronic Myelomonocytic Leukemia Myelodysplastic Syndrome Previously Treated Myelodysplastic Syndrome Recurrent Adult Acute Myeloid Leukemia Drug: Erlotinib Hydrochloride Other: Laboratory Biomarker Analysis Phase 2

Detailed Description:


I. To assess the efficacy of erlotinib (erlotinib hydrochloride) in patients with refractory or relapsed acute myeloid leukemia (AML).

II. To determine the safety and tolerability of erlotinib in this patient population.


I. To investigate inhibitory effect of this drug on spleen tyrosine kinase (SYK) and its down-stream targets such as mitogen-activated protein kinase 8 (JNK), mitogen-activated protein kinase (MAPK) and mitogen-activated protein kinase 1 (Erk).

II. To evaluate its role in janus kinase (Jak)/signal transducer and activator of transcription (STAT) pathway and to investigate erlotinib-mediated cell death and/or differentiation.

III. To quantitate concentrations of plasma erlotinib.


Patients receive erlotinib hydrochloride orally (PO) once daily (QD) on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Phase II Study of Erlotinib for the Treatment of Patients With Refractory/Relapsed AML
Actual Study Start Date : May 16, 2013
Actual Primary Completion Date : October 25, 2018
Actual Study Completion Date : October 25, 2018

Arm Intervention/treatment
Experimental: Treatment (erlotinib hydrochloride)
Patients receive erlotinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Drug: Erlotinib Hydrochloride
Given PO
Other Names:
  • Cp-358,774
  • OSI-774
  • Tarceva

Other: Laboratory Biomarker Analysis
Correlative studies

Primary Outcome Measures :
  1. Participants With a Response [ Time Frame: Up to 3 months post-treatment ]
    Overall Response is complete remission (CR) + CR with incomplete hematologic recovery (CRi) + Partial remission (PR) + Hematologic improvement (HI) + Morphologic Leukemia-Free State (MLF). (CR) is Bone marrow; </=5% blasts, no Auer rods or extramedullary disease and peripheral blood counts >/= 1.0x10^9/L Neutrophils, >/= 100x10^9/L platelets and no circulating blasts. (CRi), same as CR for bone marrow and <1.0x10^9/L neutrophils and < 100x10^9/L platelets in peripheral blood counts. PR is all CR criteria if abnormal prior to treatment except >/= 50%reduction in bone marrow blast but still > 5%. MLF is </=5% myeloblasts on bone marrow . HI response must be described by the number of positively affected cell lines..

  2. Incidence of Clinically Significant, Non-hematologic Grade 3 or 4 Toxicities at Least Possibly Related to Erlotinib Hydrochloride [ Time Frame: Up to 30 days ]
    Safety summaries will include tabulations in the form of tables and listings. The number of participants affected by treatment-emergent adverse events will be reported.

  3. Overall Survival [ Time Frame: Up to 97 weeks ]
    Time from date of treatment start until date of death due to any cause or last Follow-up.

  4. Event-free Survival [ Time Frame: Up to 21 weeks ]
    Time from date of treatment start until the date of first objective documentation of disease-relapse.

Secondary Outcome Measures :
  1. Biomarker Expressions [ Time Frame: Up to 30 days ]
    Descriptive statistics will be used to summarize the expression of biomarkers and the concentrations of plasma erlotinib hydrochloride. The Wilcoxon rank sum test will be used to compare the expressions of biomarkers and concentrations of plasma erlotinib hydrochloride between patients with and without response.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

Inclusion Criteria:

  • Patients with AML who have either been refractory to prior therapy or have relapsed after prior therapy; patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) who received therapy with a hypomethylating agent and progress to AML are eligible if they have received any therapy for MDS and failed (i.e., lack or loss of response) regardless of whether they have received therapy for AML or not; the World Health Organization (WHO) classification will be used for AML
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2
  • Total bilirubin =< 2 x upper limit of normal (ULN)
  • Alanine aminotransferase (ALT) =< 2.5 x ULN
  • Creatinine =< 2 x ULN
  • Patients must provide written informed consent
  • Patients must have been off chemotherapy for 2 weeks prior to entering this study, unless there is evidence of rapidly progressive disease, and must have recovered from the clinically significant toxic effects of that therapy to at least grade 1; use of hydroxyurea for patients with rapidly proliferative disease is allowed before the start of study therapy and for the first four weeks on therapy
  • Patients-both males and females-with reproductive potential (i.e., menopausal for less than 1 year and not surgically sterilized) must practice effective contraceptive measures throughout the study; women of childbearing potential must provide a negative pregnancy test (serum or urine) within 14 days prior to initiation of study

Exclusion Criteria:

  • Patients with known allergy or hypersensitivity to erlotinib
  • Patients with any other known disease (except carcinoma in-situ) concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes; cardiovascular disease including congestive heart failure New York Heart Association [NYHA] class III or IV, myocardial infarction within 6 months, and poorly controlled hypertension; chronic renal failure; or active uncontrolled infection) which, in the opinion of the investigator could compromise participation in the study
  • Patients unwilling or unable to comply with the protocol
  • Significant gastrointestinal disorders that may interfere with absorption of erlotinib
  • Patients who can receive a stem cell transplant within 4 weeks

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01664897

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United States, Texas
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Astellas Pharma Inc
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Principal Investigator: Jorge Cortes M.D. Anderson Cancer Center
  Study Documents (Full-Text)

Documents provided by M.D. Anderson Cancer Center:
Additional Information:
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Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01664897    
Other Study ID Numbers: 2012-0060
NCI-2012-02073 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2012-0060 ( Other Identifier: M D Anderson Cancer Center )
P30CA016672 ( U.S. NIH Grant/Contract )
First Posted: August 14, 2012    Key Record Dates
Results First Posted: January 7, 2020
Last Update Posted: January 7, 2020
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Leukemia, Myelomonocytic, Chronic
Leukemia, Myelomonocytic, Juvenile
Leukemia, Promyelocytic, Acute
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Myelodysplastic-Myeloproliferative Diseases
Erlotinib Hydrochloride
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action