Eltrombopag With or Without Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS)

Leukemia Clinical Trial

Official title: Phase II Study of Eltrombopag With or Without Continuation of Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS)

Status Completed

Clinical Trial Summary

The goal of this clinical research study is to learn if eltrombopag can help to control MDS. The safety of this drug will also be studied.

Clinical Trial Description

Study Groups:

If you are found to be eligible to take part in this study, you will be assigned to 1 of 2 arms.The selection of treatment arm will be made by you and your treating physician.

• If you are in Arm A, you will receive eltrombopag alone.

• If you are in Arm B, you will receive eltrombopag and will continue to receive the hypomethylating agent that you were receiving before you took part in this study.

Study Drug Administration:

You will take eltrombopag by mouth every day of each 28-day study cycle. If you are in Arm B, you will also continue to take the hypomethylating agent you took before joining the study at the same dosing schedule you were receiving before entering this study.

Eltrombopag should be taken on an empty stomach (1 hour before or 2 hours after a meal). Do not eat calcium-rich foods (such as dairy products and calcium fortified juices), or take other drugs (such as antacids) or supplements containing iron, calcium, aluminum, magnesium, selenium, and/or zinc for 2 hours before or 4 hours after taking eltrombopag. If a dose of eltrombopag is vomited, it should not be made up or re-taken on the same day. If the morning dose is missed, it may be taken up until 5:00 PM on the same day.

Study Visits:

On Day 1 of all Cycles:

• You will have a physical exam including vital signs.

• Blood (about 2-3 teaspoons) will be drawn for routine tests.

On Days 8, 15, and 22 of Cycle 1:

• Your vital signs (blood pressure, heart rate, and temperature) will be measured.

• Blood (about 2-3 teaspoons) will be drawn for routine tests.

If the doctor thinks it is needed, on Day 1 of every 3 cycles (Cycles 3, 6, 9, and so on), you will also have a bone marrow aspirate/biopsy to check the status of the disease and for cytogenetic testing.

Length of Study:

You may continue taking the study drug(s) for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug(s) 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 after the follow-up visits.

End-of-Treatment Visit:

Within 5 days of your last dose of study drug, you will come to the clinic for an end-of-treatment visit. The following procedures will be performed:

• You will have a physical exam.

• Blood (about 2-3 teaspoons) will be drawn for routine tests.

• You may have a bone marrow aspirate/biopsy collected to check the status of the disease and for cytogenetic testing.

Follow-up Visit:

About 28 days after your last dose of study drug, you will come to the clinic for a follow-up visit. the following procedures will be performed:

• You will have a physical exam.

• Blood (about 2-3 teaspoons) will be drawn for routine tests.

This is an investigational study. Eltrombopag is FDA approved and commercially available for the treatment of low platelet counts in patients with idiopathic thrombocytopenic purpura (ITP -- a severe bleeding disease). Its use in this study is investigational. Azacitidine and decitabine are each FDA approved for the treatment of MDS and are commercially available. The study doctor can explain how the study drug(s) are designed to work.

Up to 46 patients will take part in this study. All will be enrolled at MD Anderson. ;

Related Conditions & MeSH terms

• Leukemia
• Myelodysplastic Syndromes
• Preleukemia

• NCT number: NCT01893372
• Study type: Interventional
• Source: M.D. Anderson Cancer Center
• Status: Completed
• Phase: Phase 2
• Start date: October 2013
• Completion date: January 6, 2019