Ruxolitinib and Lenalidomide for Patients With Myelofibrosis

Myeloproliferative Diseases Clinical Trial

Official title: Evaluation of Ruxolitinib and Lenalidomide Combination as a Therapy for Patients With Myelofibrosis

Status Completed

Clinical Trial Summary

The goal of this clinical research study is to learn if the combination of ruxolitinib and lenalidomide can help to control MF. The safety of this study drug combination will also be studied.

Ruxolitinib is designed to stop certain proteins (called JAK1 and JAK2) that are found in MF cells from sending signals that may lead to the growth of cancer cells.

Lenalidomide is designed to change the body's immune system. It may also interfere with the development of tiny blood vessels that help support tumor growth. This may decrease the growth of cancer cells.

Clinical Trial Description

Study Drug Administration:

If you are found eligible to take part in this study, you will begin taking ruxolitinib by mouth 2 times every day during each 28-day cycle. You should take ruxolitinib one time in the morning and one time in the evening (about 12 hours apart). Ruxolitinib should be taken with a meal and a glass of water. If you miss a dose of ruxolitinib, you should not make up the dose or take any more before the next scheduled dose.

You will also take lenalidomide by mouth one time in the morning on Days 1-21 of each cycle. Lenalidomide capsules should be swallowed whole, and should not be broken, chewed, or opened. If a dose of lenalidomide is missed, it should be taken as soon as possible on the same day. If it is missed for the entire day, it should not be made up.

If your study doctor thinks it is needed or if you have side effects, your dose of ruxolitinib and/or lenalidomide may be stopped, lowered, or delayed for up to 8 weeks during the study.

During the study, you will need to return all unused study drug to the study staff at each clinic visit.

Additional Drugs:

If your disease has not responded to the study drugs after 3 cycles and the study doctor thinks it is in your best interest, you may begin taking prednisone along with the study drugs. Prednisone is a corticosteroid that is similar to a natural hormone made by your body. Prednisone is often given in combination with other chemotherapy drugs. You will take prednisone every morning during Cycles 4 and 5, then every other day during Cycle 6. After Cycle 6, you will no longer take prednisone. If you forget to take a does of prednisone and more than 8 hours have passed, wait until the next day to take prednisone again (or 2 days later if you are taking it every other day during Cycle 6).

If your doctor thinks it is needed, you may take aspirin during this study to help prevent blood clots from forming. If you are allergic to aspirin or cannot take aspirin, your doctor may recommend you take another type of drug to help prevent blood clots from forming.

Study Drug Diary:

You will be given a study drug diary before you begin taking the study drugs to write down what time you take each dose of the study drugs. You will need to bring the diary with you to each study visit so it can be reviewed.

You should bring the study drug (including empty bottles) with you to all of the study visits. You will be asked not to take your morning dose of study drug before your visits on Day 15 Cycle 1 and Day 1 on Cycle 2.

Study Visits:

On Day 1 of Cycles 1 and 2:

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

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

• Blood (about 3 teaspoons) will be drawn for routine tests and to check your kidney and liver function. This routine blood draw will include a pregnancy test if you are able to become pregnant.

• You will complete 2 questionnaires about your quality of life. The questionnaires should take about 10 minutes to complete. One of the questionnaires may be filled out on a computer. The study staff will help you use the computer.

On Days 8 and 22 of Cycles 1 and 2:

° Blood (about 3 teaspoons) will be drawn for routine tests. This routine blood draw will include a pregnancy test if you are able to become pregnant.

On Day 15 of Cycles 1 and 2:

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

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

• Blood (about 3 teaspoons) will be drawn for routine tests and to check your kidney and liver function. This routine blood draw will include a pregnancy test if you are able to become pregnant.

On Day 1 of Cycle 3 and every 3 to 6 Cycles after that:

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

• You will have a review of any blood transfusions you may have had in the last 3 months.

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

• Blood (about 3 teaspoons) will be drawn for routine tests and to check your kidney and liver function. This routine blood draw will include a pregnancy test if you are able to become pregnant.

• You will complete 2 questionnaires about your quality of life. The questionnaires should take about 10 minutes to complete.

You will have a bone marrow biopsy and aspirate after Cycles 3, 6, 9, and 12, and then when your doctor feels it is necessary.

Interactive Voice Response (IVR) System:

During the first 3 cycles, you will use the Interactive Voice Response (IVR) system once a month to tell the study staff about any side effects you may be having. The IVR system is an automated calling system that will allow you to press buttons on your phone to answer questions about any side effects you may be having. The study staff will give you instructions on how to use the IVR. If you are not able to use the IVR system, a member of the study staff will contact you instead.

After Cycle 3, a member of the study staff will call you at your home once a month to ask you about any side effects you may be having and to review the results of your blood tests.

Length of Study:

You may receive the study drug combination for up to 6 years. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

You will be considered off study after your follow-up visit.

End-of-Study Visit:

You will have an end-of-study visit the day you are taken off study. The following tests and procedures will be performed:

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

• You will have a review of any blood transfusions you may have had in the last 3 months.

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

• Blood (about 3 teaspoons) will be drawn for routine tests and to check your kidney and liver function. This routine blood draw will include a pregnancy test if you are able to become pregnant.

• You will complete 2 questionnaires about your quality of life. The questionnaires should take about 10 minutes to complete.

• You will have a bone marrow biopsy and/or aspirate to check the status of the disease.

Follow-up:

A member of the study staff will call you 30 days and 60 days after you have stopped taking the study drugs to ask how you are feeling and if you have had any side effects since your last visit.

This is an investigational study. Lenalidomide is not FDA-approved or commercially available for use in patients with MF. Ruxolitinib is FDA approved and commercially available for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocythemia (post-ET) myelofibrosis. Lenalidomide is FDA-approved for the treatment of some forms of myelodysplastic syndrome (MDS) and multiple myeloma (MM). Its use in patients with MF is investigational.

Up to 49 patients will take part in this study. All will be enrolled at MD Anderson. ;

Related Conditions & MeSH terms

• Myeloproliferative Diseases
• Myeloproliferative Disorders
• Primary Myelofibrosis

• NCT number: NCT01375140
• Study type: Interventional
• Source: M.D. Anderson Cancer Center
• Status: Completed
• Phase: Phase 2
• Start date: September 22, 2011
• Completion date: September 5, 2018