MEK Inhibitor 162 Relapsed and/or Refractory Acute Myeloid Leukemia (AML) and Poor Prognosis, Not Suitable for or Unwilling to Receive Standard Therapy

Leukemia Clinical Trial

Official title: Phase I/II Trial of MEK Inhibitor MEK162 in Patients With Relapsed and or Refractory Acute Myeloid Leukemia and Patients With Poor Prognosis Acute Myeloid Leukemia Not Suitable for or Unwilling to Receive Standard Therapy

Status Terminated

Clinical Trial Summary

The goal of Phase 1 of this clinical research study is to find the highest tolerable dose of MEK162 that can be given to patients with advanced leukemia.

This is an investigational study. MEK162 is not FDA approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work.

Up to 57 patients total will take part in both phases of this study . All will be enrolled at MD Anderson.

The goal of Phase 2 of this clinical research study is to learn if MEK162 can help to control AML in older patients with advanced leukemia. The safety of this drug will also be studied.

This is an investigational study. MEK162 is not FDA approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work.

Up to 57 patients total will take part in both phases of this study. All will be enrolled at MD Anderson.

Clinical Trial Description

Phase I:

Study Groups:

If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 2 groups of 3-6 participants will be enrolled in Phase 1 of the study.

If you are enrolled in Phase 1, the dose of MEK162 you receive will depend on when you joined this study. The first group of participants will receive the lowest dose level of MEK162. The second group will receive a higher dose of MEK162 than the group before it, if no intolerable side effects were seen. This is designed to find the highest tolerable dose of MEK 162.

Participants in Phase 2 will then receive the highest tolerated dose that was found in Phase 1.

Study Drug Administration:

You will take MEK162 tablets by mouth 2 times a day (about 12 hours apart) every day with a full cup of water.

There are 4 weeks in each study cycle.

Study Visits:

On Day 1 of Cycle 1:

• You will have a physical exam.

• Blood (about 4 teaspoons) will be drawn for routine tests and blood sugar testing.

On Days 4 and 8 of Cycle 1, blood (about 4 teaspoons) will be drawn for routine tests and blood sugar testing.

Sometime during Days 13-27 of each cycle and again on Day 28 of Cycles 2 and beyond, the study staff will call you and ask about any drugs you may be taking and any side effects you may be having. The calls should last less than 10 minutes each time.

On Day 28 of Cycle 1, you will have a bone marrow aspirate and/or biopsy to check the status of the disease and for biomarker and cytogenetic testing. If bone marrow cannot be collected, blood (about 1-2 teaspoons) will be drawn for these tests.

You will have an EKG and either an ECHO or MUGA scan to check your heart function.

On Day 1 of Cycle 2 and beyond:

• You will have a physical exam.

• Blood (about 4 teaspoons) will be drawn for routine tests and blood sugar testing.

• Urine will be collected for routine tests.

• You will have an eye exam by an eye doctor.

• If you can become pregnant, blood (about 1 teaspoon) or urine will be collected for a pregnancy test.

On visits when you have blood sugar testing, you should fast for at least 8 hours before the blood draw.

On visits when you have urine collected, you will be given containers to collect your urine over 24 hours if the doctor thinks it is needed.

If it is more convenient to you, you may be able to have some of your blood draws done away from MD Anderson. The study staff will discuss this with you.

Every two or three months during your treatment, you will have an echocardiogram (ECHO) or a multigated acquisition (MUGA) scan to check your heart function.

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:

After your last dose of study drug:

• You will have a physical exam.

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

• If the doctor thinks it is needed, you will have a bone marrow aspirate and/or biopsy to check the status of the disease and for biomarker, PD, and/or cytogenetic testing. If you have already had a bone marrow aspirate and/or biopsy recently and your doctor thinks repeating it is not needed, leftover bone marrow will be used for these tests. If bone marrow cannot be collected, blood (about 1-2 teaspoons) will be drawn for these tests.

• You will have an EKG.

Phase II:

Study Groups:

If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 42 participants will be enrolled in Phase 2 of the study.

Participants in Phase 1 were given a dose of MEK162 based on when they joined the study. Groups of participants were given different dose levels until the highest tolerable dose of MEK162 was found.

If you are enrolled in Phase 2, you will receive MEK162 at the highest dose that was tolerated in Phase 1.

Study Drug Administration:

You will take MEK162 tablets by mouth 2 times a day (about 12 hours apart) every day with a full cup of water.

There are 4 weeks in each study cycle.

Study Visits:

On Day 1 of Cycle 1:

• You will have a physical exam.

• Blood (about 4 teaspoons) will be drawn for routine tests and blood sugar testing.

On Days 4 and 8 of Cycle 1, blood (about 4 teaspoons) will be drawn for routine tests and blood sugar testing.

Sometime during Days 13-27 of each cycle and again on Day 28 of Cycles 2 and beyond, the study staff will call you and ask about any drugs you may be taking and any side effects you may be having. The calls should last less than 10 minutes each time.

On Day 28 of Cycle 1, you will have a bone marrow aspirate and/or biopsy to check the status of the disease and for biomarker and cytogenetic testing. If bone marrow cannot be collected, blood (about 1-2 teaspoons) will be drawn for these tests.

You will have an EKG and either an ECHO or MUGA scan to check your heart function.

On Day 1 of Cycle 2 and beyond:

• You will have a physical exam.

• Blood (about 4 teaspoons) will be drawn for routine tests and blood sugar testing.

• Urine will be collected for routine tests.

• You will have an eye exam by an eye doctor.

• If you can become pregnant, blood (about 1 teaspoon) or urine will be collected for a pregnancy test.

On visits when you have blood sugar testing, you should fast for at least 8 hours before the blood draw.

On visits when you have urine collected, you will be given containers to collect your urine over 24 hours if the doctor thinks it is needed.

If it is more convenient to you, you may be able to have some of your blood draws done away from MD Anderson. The study staff will discuss this with you.

Every two or three months during your treatment, you will have an echocardiogram (ECHO) or a multigated acquisition (MUGA) scan to check your heart function.

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:

After your last dose of study drug:

• You will have a physical exam.

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

• If the doctor thinks it is needed, you will have a bone marrow aspirate and/or biopsy to check the status of the disease and for biomarker, PD, and/or cytogenetic testing. If you have already had a bone marrow aspirate and/or biopsy recently and your doctor thinks repeating it is not needed, leftover bone marrow will be used for these tests. If bone marrow cannot be collected, blood (about 1-2 teaspoons) will be drawn for these tests.

• You will have an EKG. ;

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute

• NCT number: NCT02089230
• Study type: Interventional
• Source: M.D. Anderson Cancer Center
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: August 27, 2014
• Completion date: June 5, 2019