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A Phase II Trial of Eltrombopag for Patients With Chronic Lymphocytic Leukemia (CLL) and Thrombocytopenia

Phase 2
18 Years
Open (Enrolling)
CLL, Leukemia

Thank you

Trial Information

A Phase II Trial of Eltrombopag for Patients With Chronic Lymphocytic Leukemia (CLL) and Thrombocytopenia

The Study Drug:

When the number of platelets in your body gets too low, it can cause bleeding, which may
cause serious health problems and/or prevent you from receiving chemotherapy. Eltrombopag
is designed to act like a protein in your body that helps make platelets. This may help
increase your platelet counts.

Study Drug Administration:

You will take pills of the study drug by mouth 1 time each day on an empty stomach (1 hour
before or 2 hours after a meal). You should take the pills with 1 cup (8 ounces) of water.
You should wait at least 4 hours between taking eltrombopag and eating foods with calcium
(dairy products and/or juices with added calcium) or taking drugs/supplements with iron,
calcium, aluminum, magnesium, selenium and/or zinc. Other drugs may also affect eltrombopag.
Be sure to tell your doctor about any drugs and/or supplements you may be taking.

During your study visits, your doctor will check your platelet counts to see if they
improve. If they do not improve, your dose of study drug may be increased. Your doctor
will instruct you on each dose of eltrombopag you should take.

Do not take more than 1 dose of eltrombopag on any one day. If you forget to take a dose,
you should skip the missed dose and continue your regular dosing schedule. Do not take a
double dose to make up for a missed one.

Study Visits:

Each study "cycle" will be 28 days.

Each week during Cycle 1, then every 2 weeks during Cycles 2 and 3:

- You will have a physical exam, including measurement of your vital signs.

- You will be asked about any bleeding that may have occurred since the last study visit.

- Blood (about 1 tablespoon) will be drawn for routine tests.

On Day 1 (+/- 7 days) of Cycles 4 and beyond:

- You will have a physical exam.

- You will be asked about any bleeding that may have occurred since the last study visit.

- Blood (about 1 tablespoon) will be drawn for routine tests.

- You will also have a bone marrow aspirate/biopsy to check the status of the disease and
to check your platelet counts. This test will only be performed every 3 cycles.

Length of Study Participation:

You may continue taking the study drug for as long as the doctor thinks it is in your best
interest. You will no longer be able to take the study drug if you require other treatment
for CLL or if intolerable side effects occur.


After you stop taking eltrombopag for any reason, your platelet counts may drop. This may
increase your risk of bleeding. Blood (about 1 tablespoon) will be drawn each week for 4
weeks to check your platelet counts.

This is an investigational study. Eltrombopag is FDA approved and commercially available
for use in chronic immune thrombocytopenic purpura (ITP - severe bleeding due to platelet
destruction by the immune system). The use of this drug in patients with CLL is

Up to 36 patients will take part in this study. All will be enrolled at MD Anderson.

Inclusion Criteria:

1. Diagnosis of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)

2. Age >/= 18 years

3. PLT transfusion-dependent, defined as need for transfusion to maintain PLT count
>/=20K/µL, or the average of two (non-transfused) PLT counts taken within 2 weeks of
the screening period 55K/µL

4. Patients with ITP must have failed at least 1 prior treatment for ITP including one
of the following: corticosteroids, rituximab, splenectomy, cyclosporine

5. At least 3 weeks must have elapsed since the last chemotherapy treatment for CLL

6. ECOG performance status (PS)
7. Adequate liver function (total bilirubin ULN)

8. Adequate renal function (serum creatinine Cr
9. For patients with ITP on corticosteroids or cyclosporine, dose of corticosteroids or
cyclosporine must be stable for 2 weeks prior to enrollment and planned to be tapered
in patients responding to eltrombopag

10. Able to provide informed consent

Exclusion Criteria:

1. Concurrent chemotherapy for CLL

2. Diagnosis of Richter's transformation

3. Uncontrolled autoimmune hemolytic anemia i.e. patients with AIHA that is not
controlled with treatment such as corticosteroids or cyclosporine. This would include
patients who require PBRC transfusions or who do not have a stable hemoglobin (HGB)
due to ongoing hemolysis.

4. Concurrent treatment for ITP (except for corticosteroids and cyclosporine)

5. Diagnosis of myelodysplastic syndrome or acute myeloid leukemia

6. Active infection or significant medical illness as determined by the treating

7. Treatment with thrombomimetic agents in the past 3 months (rTPO, PEG-rHuMGDF, Nplate
or Promacta)

8. Pregnant or breast feeding subjects and subjects not willing to use adequate
contraceptive precautions

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 Patients with Overall Response

Outcome Description:

Overall response rate (OR), which is defined as the composite of complete response (CR) and major response (MR).

Outcome Time Frame:

3 Months

Safety Issue:


Principal Investigator

William G. Wierda, MD, PHD, BS

Investigator Role:

Study Chair

Investigator Affiliation:

UT MD Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

November 2010

Completion Date:

Related Keywords:

  • CLL
  • Leukemia
  • Chronic Lymphocytic Leukemia
  • CLL
  • Thrombocytopenia
  • Small lymphocytic lymphoma
  • SLL
  • Eltrombopag
  • Promacta
  • thrombopoietin (TPO)-receptor agonist
  • platelets
  • Leukemia
  • Leukemia, Lymphocytic, Chronic, B-Cell
  • Leukemia, Lymphoid
  • Thrombocytopenia



UT MD Anderson Cancer Center Houston, Texas  77030