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Phase II Study Evaluating the Role of Erlotinib an Epidermal Growth Factor Receptor (EGFR) Inhibitor in the Treatment of Myelodysplastic Syndrome

Phase 2
18 Years
Not Enrolling
Myelodysplastic Syndrome

Thank you

Trial Information

Phase II Study Evaluating the Role of Erlotinib an Epidermal Growth Factor Receptor (EGFR) Inhibitor in the Treatment of Myelodysplastic Syndrome

Screening Period: Informed consent, physical examination, medical history report, blood
tests, pregnancy test (if applicable), list of current medications, description of symptoms,
chest x-ray, ECG, bone marrow aspirate/biopsy within 4 weeks of study start.

Weeks 2,6,10 and 14: Blood tests.

Weeks 4 and 12: Blood tests, physical exam, patients will answer question about how they are
feeling and if there are any changes to medication they have taken.

Weeks 8 and 16: Blood tests, physical exam, patients will answer question about how they are
feeling and if there are any changes to medication they have taken, bone marrow
aspirate/biopsy (if physician has determined the patient has had a clinical response or
partial response to treatment.

After week 16 (if responding to treatment): Have a bone marrow aspirate/biopsy (will be
repeated at time of relapse, i.e., more than 50% increase in the percentage of myeloblasts
[leukemia cells] or drop in blood counts after they improved or requiring regular blood
transfusions after not requiring them for at least 8 weeks,or after 1 year in study).

After the patient has stopped taking erlotinib: Periodic follow-up on patients' status.

Inclusion Criteria:

- Patients must have an established diagnosis of myelodysplastic syndrome (MDS) and
have either: Low or intermediate 1 risk disease by IPSS with symptomatic anemia
(defined as hemoglobin less than 10.0 g/dl) or transfusion dependent anemia (defined
as requiring ≥ 4 units of blood RBCs administered with a pretreatment hemoglobin
value of ≤ 9 g/dL in the 8 weeks prior to Day 1 of treatment in this study). Patients
with anemia must have no response to at least to 6 weeks trial of erythroid
stimulating agents (ESA) [erythropoietin/ darbepoetin]. Patients with serum
erythropoietin levels more than 500 mU/ ml on diagnosis are eligible to the study
without erythropoietin/darbepoetin prior treatment. Patients who do not meet anemia
criteria are still eligible if they had thrombocytopenia with two or more platelet
counts < 50 x 10^9/L or a significant clinical hemorrhage requiring platelet
transfusions or if they had neutropenia with an ANC < 1 x 10^9/L; Intermediate-2 or
high risk MDS by IPSS.

- Patients ≥ 60 years with AML by WHO classification and myeloblasts percentage 20-30%
(RAEB-t by MDS FAB classification) are eligible for the study if deemed not suitable
for induction chemotherapy or declined that option.

- All prior treatment must have been discontinued 28 days prior to Day 1 of treatment
in this study except (ESA) and colony stimulating factors where it should be stopped
14 days prior to start therapy on study, and hydroxyurea should be stopped 2 days

- Prior bone marrow or stem cell transplant is allowed.

- Secondary or therapy related MDS patients are eligible.

- Patients with chronic myelomonocytic leukemia (CMML) are eligible.

- Patients must have a performance status of 0 - 2 by Zubrod performance status

- Pretreatment pathology materials must be available for morphologic review. Collection
of blood and marrow specimens for pathology review must be completed within 28 days
prior to registration.

- No other prior malignancy is allowed except for the following: adequately treated
basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated
Stage I or II cancer from which the patient is currently in complete remission, or
any other cancer from which the patient has been disease-free for at least 2 years.

- In calculating days of tests and measurements, the day a test or measurement is done
is considered Day 0. Therefore, if a test is done on a Monday, the Monday four weeks
later would be considered Day 28. This allows for efficient patient scheduling
without exceeding the guidelines. If Day 28 or 60 falls on a weekend or holiday, the
limit may be extended to the next working day.

- All patients must be informed of the investigational nature of this study and must
sign and give written consent in accordance with institutional and federal

Exclusion Criteria:

- Patients must not have received prior remission induction chemotherapy as treatment
for MDS.

- Patients must not be pregnant or nursing because of the potential risks of the drugs
used in this study. Women/men of reproductive potential may not participate unless
they have agreed to use an effective contraceptive method.

- Patients who are known HIV positive are not eligible for this study.

Type of Study:


Study Design:

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

Outcome Measure:

Number of Participants With Overall Response (OR)

Outcome Description:

To test whether Erlotinib is sufficiently effective in the treatment of myelodysplastic syndrome (MDS) to warrant further investigation. The primary measure of effectiveness is the overall response rate using International working group criteria (IWG).

Outcome Time Frame:

21 Months

Safety Issue:


Principal Investigator

Rami Komrokji, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

H. Lee Moffitt Cancer Center and Research Institute


United States: Institutional Review Board

Study ID:




Start Date:

September 2009

Completion Date:

June 2012

Related Keywords:

  • Myelodysplastic Syndrome
  • MDS
  • blood diseases
  • bone marrow
  • hematopoietic
  • leukemia
  • Myelodysplastic Syndromes
  • Preleukemia



H. Lee Moffitt Cancer Center & Research Institute Tampa, Florida  33612