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A Pilot Phase II Study of Erlotinib for the Treatment of Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)

Phase 2
18 Years
Open (Enrolling)

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Trial Information

A Pilot Phase II Study of Erlotinib for the Treatment of Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)

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

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

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.

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

2. Age >/=18 years

3. ECOG Performance Status
4. Adequate liver (total bilirubin
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

5. Patients who can receive a stem cell transplant within 4 weeks.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall Response Rate (ORR)

Outcome Description:

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).

Outcome Time Frame:

28 days

Safety Issue:


Principal Investigator

Jorge Cortes, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

May 2013

Completion Date:

Related Keywords:

  • Leukemia
  • Leukemia
  • Acute myeloid leukemia
  • AML
  • refractory
  • relapsed
  • Erlotinib
  • Erlotinib Hydrochloride
  • OSI-774
  • CP358774
  • Tarceva
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid



University of Texas MD Anderson Cancer CenterHouston, Texas  77030