Pilot Phase II - Erlotinib for Acute Myeloid Leukemia (AML)

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 : Active, not recruiting
First Posted : August 14, 2012
Last Update Posted : March 20, 2018
Astellas Pharma Inc
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
The goal of this clinical research study is to learn if erlotinib can help to control AML. The safety of this drug will also be studied.

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

Detailed Description:

Study Drug Administration:

Each cycle is 28 days.

You will take erlotinib by mouth 1 time each day.

If you have severe side effects from the study drug, the study doctor may decide to stop your drug dosing until the side effects get better.

Study Visits:

Every week for the first 3 months and then every 2-4 weeks after that, blood (about 1 tablespoon) will be drawn for routine tests and to test your liver and kidney function. After 6 months, blood will be drawn every 6-8 weeks.

At the end of Cycle 1 and then every 2-3 months after that, you will have a complete physical exam, and you will be asked about any side effects you may have or any drugs you are taking.

At the end of Cycle 1 and then every 2-3 months after that for 1 year, you will have a bone marrow aspirate. After 1 year, you will only have additional bone marrow aspirates performed if the study doctor thinks it is necessary.

If you have no significant side effects during the first 12 months, the number of blood draws and bone marrow collections may be lowered to 1 every 1-3 cycles.


You may continue taking the study drug for as long as the study doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

End-of-Treatment Visit:

You will have an end-of-treatment visit 30 days (+/- 7 days) after your last dose of erlotinib.

At this visit, you will be asked about any symptoms and/or side effects you may have. The end-of-treatment visit can be done by phone or during one of your standard of care clinic visits at MD Anderson. If this visit is done by phone, the call should last about 15 minutes.

This is an investigational study. Erlotinib is not FDA approved and commercially available for the treatment of AML. In this disease type, it is currently being used for research purposes only.

Up to 29 patients will take part in this study. All will be enrolled at MD Anderson.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 29 participants
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 Acute Myeloid Leukemia (AML)
Actual Study Start Date : May 2013
Estimated Primary Completion Date : May 2020
Estimated Study Completion Date : May 2021

Arm Intervention/treatment
Experimental: Erlotinib

Patients receive therapy with erlotinib administered orally as a continuous daily dose in 28-day cycles. Patients will continue therapy until clinically significant progression of the disease or unacceptable toxicity.

Erlotinib starting dose 150 mg by mouth, once daily in 28-day cycles.

Drug: Erlotinib
Starting dose: 150 mg by mouth once daily in a 28-day cycle.
Other Names:
  • Erlotinib Hydrochloride
  • OSI-774
  • CP358774
  • Tarceva

Primary Outcome Measures :
  1. Overall Response Rate (ORR) [ Time Frame: 28 days ]
    Primary efficacy endpoint is overall response (ORR) as assessed by investigators. An adequate response, included in ORR for this trial will include all of the following obtained within the first 3 months of therapy: complete remission (CR), complete remission with incomplete blood count recovery (CRi), partial remission (PR), and morphologic leukemia-free state (MLF).

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.

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

Inclusion Criteria:

  1. Patients with AML who have either been refractory to prior therapy or have relapsed after prior therapy. Patients with MDS or 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 WHO classification will be used for AML.
  2. Age >/=18 years
  3. ECOG Performance Status </=2
  4. Adequate liver (total bilirubin </=2x ULN, ALT </=2.5x ULN) and renal (creatinine </=2x ULN) function.
  5. Patients must provide written informed consent.
  6. 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.
  7. Patients - both males and females - with reproductive potential (ie, 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:

  1. Patients with known allergy or hypersensitivity to erlotinib.
  2. 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 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.
  3. Patients unwilling or unable to comply with the protocol.
  4. Significant gastrointestinal disorders that may interfere with absorption of erlotinib.
  5. 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

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Astellas Pharma Inc
Principal Investigator: Jorge Cortes, MD M.D. Anderson Cancer Center

Additional Information:
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01664897     History of Changes
Other Study ID Numbers: 2012-0060
NCI-2012-02073 ( Registry Identifier: NCI CTRP )
First Posted: August 14, 2012    Key Record Dates
Last Update Posted: March 20, 2018
Last Verified: March 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by M.D. Anderson Cancer Center:
Acute myeloid leukemia
Erlotinib Hydrochloride

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Erlotinib Hydrochloride
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action