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Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML)


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Leukemia

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Trial Information

Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML)


Study Drug Administration:

If you are found to be eligible to take part in this study, you will receive eltrombopag by
mouth 1 time a day. Your dose may be increased every 2 weeks depending on your platelet
count response.

You should take eltrombopag on an empty stomach. You should not eat for 2 hours before
taking eltrombopag. You should wait at least 4 hours between taking eltrombopag and taking
other drugs (like antacids), dairy products, juices with calcium added, or supplements
containing iron, calcium, aluminum, magnesium, selenium, or zinc.

Study Visits:

Some of the routine blood draws listed below may be done at a clinic near your home. Ask
the study staff for more details about this.

The dose adjustment phase is when the study doctor will change the dose of the study drug or
your drug for treatment of CML or myelofibrosis.

During the dose adjustment phase:

- Before the most effective dose is found, blood (about 1-2 tablespoons) will be drawn 1
time every week for routine tests and to check your platelet counts.

- After the most effective dose is found, blood (about 1-2 tablespoons) will be drawn 1
time each month for routine tests and to check your platelet counts.

During the first year of eltrombopag therapy, every 3-4 months:

- Your medical history will be recorded.

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

- You will be asked about side effects you may be having and any other drugs you may be
taking.

- You will have a standard-of-care bone marrow aspiration and/or biopsy to check the
status of the disease. This sample will also be used for genetic research tests. If
an aspirate cannot be collected, blood (about 1 teaspoon) will be drawn for these
tests.

After the first year of eltrombopag therapy, every 6 months, you will return to the clinic
and have the same tests and procedures performed as listed above.

Length of Study:

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 the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.

Your participation on the study will be over once you have completed the end-of-treatment
visit.

End-of-Treatment Visit:

About 30 days after your last dose of study drug, you will return to the clinic for an
end-of-treatment visit.

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

- You will be asked about any side effects you may be having.

- Blood (about 1-2 tablespoons) will be drawn for routine tests. This blood will also be
used to check your platelet counts.

This is an investigational study. Eltrombopag is FDA approved and commercially available
for the treatment of patients with low platelet counts. The use of eltrombopag for the
treatment of low platelet counts in patients with CML and myelofibrosis is investigational.

Up to 39 patients will take part in this study. All will be enrolled at MD Anderson.


Inclusion Criteria:



1. CML patients in chronic phase receiving treatment with any FDA approved TKI
(imatinib, nilotinib, or dasatinib); or CML patients in accelerated or blastic phase
who are considered to be in this phase because of thrombocytopenia or because of
clonal evolution and with no other criteria for accelerated/blastic phase or patients
with myelofibrosis receiving treatment with FDA approved TKI (ruxolitinib) and with
peripheral blood and/or bone marrow blasts
2. Grade >/= 3 thrombocytopenia (platelets < 50 x 10^9/L) after the first 3 months of
therapy with the TKI for patients with CML and platelets <100 x 10^9/L for patients
with MF after the first 3 months of therapy. Thrombocytopenia must be either
recurrent (i.e., second or greater episode of thrombocytopenia) or having required
dose reductions of the TKI.

3. Subject is anticipated to have therapy with TKI continued for >/= 3 months

4. Adequate organ function: Total bilirubin (except for Gilbert's Syndrome) ULN; ALT and AST < 3 x ULN; Creatinine
Exclusion Criteria:

1. CML patients in accelerated or blastic phase except for those who are considered to
be in this phase because of thrombocytopenia or because of clonal evolution and with
no other criteria for accelerated/blastic phase; or myelofibrosis patients who have
transformed to acute leukemia or have >/= 10% blasts in peripheral blood and/or in
bone marrow.

2. Thrombocytopenia that is considered to be unrelated to treatment with TKI or
accelerated phase as defined above;

3. Age < 18 years;

4. Stem cell transplantation within preceding 60 days prior to registration;

5. Patients with documented active hepatitis B or C infection;

6. Patients with known bone marrow reticulin fibrosis (only applicable to patients with
CML);

7. Patients with palpable splenomegaly >/= 16cm below coastal margin (only applicable to
patients with CML).

8. Female subjects who are pregnant or breastfeeding. Women of childbearing potential
are required to have a BHCG serum or urine pregnancy test performed within 7 days
prior to first study drug dose. A female of childbearing potential is a sexually
mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or
2) has not been naturally postmenopausal for at least 12 consecutive months (i.e.,
has had menses at any time in the preceding 12 consecutive months). Women of
child-bearing potential and men must agree to use contraception prior to study entry
and for the duration of study participation.

9. Patients with known risk factors for thromboembolism (e.g. Factor V Leiden mutation,
ATIII deficiency, Protein C and S deficiency, antiphospholipid syndrome, portal
hypertension, etc.)

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Number of Participants with Complete Platelet Response

Outcome Description:

Complete (platelet) response defined as a sustained (3 months) platelet count of ≥ 50 x 109/L and at least a 20% increase in platelet count from baseline with no more than 3 counts in that period being ≤ 50 * 109/L , while continuing imatinib or other TKI therapy. Complete Blood Counts (CBCs), including platelet count, weekly at beginning of treatment or with dose adjustments until stable platelet count achieved, monthly thereafter.

Outcome Time Frame:

Every 3 to 4 months (Baseline to 3 months confirmed Complete Platelet Response, up to 18 months following treatment initiation.)

Safety Issue:

Yes

Principal Investigator

Gautam Borthakur, MBBS

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

2011-0319

NCT ID:

NCT01428635

Start Date:

January 2012

Completion Date:

Related Keywords:

  • Leukemia
  • Leukemia
  • Thrombocytopenia
  • Tyrosine kinase therapy
  • TKI
  • Chronic myeloid leukemia
  • CML
  • Platelet count
  • Clonal evolution
  • Eltrombopag
  • Promacta
  • Leukemia
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Thrombocytopenia

Name

Location

UT MD Anderson Cancer CenterHouston, Texas  77030