Clinical trial NCT01251965
Conditions: Leukemia
Keywords: Relapsed Acute Leukemia Acute myeloid leukemia AML Acute lymphocytic leukemia ALL Ruxolitinib Jakafi INC424 INCB018424
Sponsor: M.D. Anderson Cancer Center
Start date: 2010-12-01; Completion date: 2012-07-01
Enrollment: 27 participants; Allocation: NON_RANDOMIZED; Intervention model: SINGLE_GROUP

Brief summary:
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with acute leukemia and to learn if the study drug can help control the disease. The safety of the drug will also be studied.

Detailed description:
The Study Drug:

Ruxolitinib is designed to block a gene mutation that may be important in cancer cell growth and survival. By blocking the gene mutation, this may cause the cancer cells to die.

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 30 participants will be enrolled in the Phase I portion of the study, and up to 136 participants will be enrolled in Phase II.

If you are enrolled in the Phase I portion, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose level of ruxolitinib. Each new group will receive a higher dose of ruxolitinib than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of ruxolitinib is found.

If you are enrolled in the Phase II portion, you will receive ruxolitinib at the highest dose that was tolerated in the Phase I portion or at a lower dose.

Study Drug Administration:

You will take ruxolitinib tablet(s) by mouth 2 times a day on Days 1-28 of each 28-day study cycle.

You will be asked to keep a diary to record the doses taken. You will be asked to bring your diary and any unused drug to your next visit.

Study Visits:

On Days 1, 7, 14, and 21 of Cycle 1:

* You will have a physical exam.
* Blood (about 2 teaspoons) will be drawn for routine tests.
* You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
* On Day 14 only, you will have a bone marrow aspiration performed to check the status of the disease.

On Day 1 of Cycle 2:

* You will have a physical exam.
* You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
* Blood (about 2 teaspoons) will be drawn for routine tests.
* You will have a bone marrow aspiration performed to check the status of the disease.

During Cycles 2 and beyond, blood (about 2 teaspoons) will be drawn for routine tests at least every 1-2 weeks. This blood may be drawn at a clinic close to your home.

On Day 1 of Cycles 3, 6, 9, and beyond:

* You will have a physical exam.
* You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
* Blood (about 2 teaspoons) will be drawn for routine tests.
* You will have a bone marrow aspiration performed to check the status of the disease. On Day 1 of Cycle 3, this will only be done if your doctor thinks it is needed.

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 or intolerable side effects occur.

Your participation on the study will be over once you have completed the end-of-study visit and the follow-up call.

End-of-Study Visit:

After your last dose of study drug, you will have an end-of-study visit. At this visit, the following tests and procedures will be performed:

* You will have a physical exam.
* You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
* Blood (about 2 teaspoons) will be drawn for routine tests.
* You will have a bone marrow aspiration performed to check the status of the disease.

Follow-Up:

About one month after your end-of-study visit, the study staff will call and ask about any side effects you may be having. This call should last about 5 minutes.

This is an investigational study. 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. Its use to treat acute leukemia is investigational.

Up to 166 patients will take part in this study. All will be enrolled at MD Anderson.

Arms: arm 1: Phase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle. arm 2: Phase I dose of Ruxolitinib 100 mg by mouth twice a day for 28 day cycle. arm 3: Phase I dose of Ruxolitinib 200 mg by mouth twice a day for 28 day cycle.
Interventions: intervention 1: Ruxolitinib
Intervention descriptions: intervention 1: Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle.

Phase II - MTD reached in Phase I.

Protocol documents: