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Hematologic Diseases

Myeloid Leukemia

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Phase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle.

Phase I dose of Ruxolitinib 100 mg by mouth twice a day for 28 day cycle.

Phase I dose of Ruxolitinib 200 mg by mouth twice a day for 28 day cycle.

Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle.

Phase II - MTD reached in Phase I.

Ruxolitinib

Srdan Verstovsek, MD

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.

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.

Phase I is a standard 3+3 design and will be used to determine the dose limiting toxicity (DLT) and maximum tolerated dose (MTD), and no formal evaluation of efficacy (response rate will be implemented. Patients in Phase I part who were treated at the MTD will be included toward patients assessed for response in the phase II stage.

Phase l/II Study of Ruxolitinib for Acute Leukemia

A Phase I/II Study to Determine the Safety and Efficacy of Ruxolitinib, a JAK1/JAK2 Inhibitor, in Subjects With Relapsed or Refractory Acute Leukemia

MTD defined as highest dose level at which no more than one out of six subject experiences dose limiting toxicity (DLT) during first cycle (28 days) of therapy. A non-hematologic DLT defined as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value (according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria) assessed as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during first 28 days on study. Participants who received at least 80% of the originally assigned doses in the first cycle were evaluable for DLT assessment of each cohort.

The MTD is defined as the highest dose level at which no more than one out of six subject experiences DLT during the first cycle (28 days) of therapy.

Response is defined as complete remission (CR) + complete remission with incomplete blood count (CRi) + Hematologic improvement (HI). Response was to be assessed for participants who were evaluated for the Phase II portion of this study. CR is absolute neutrophil count (ANC) \>/= 1x109/L and platelet count \>/= 100x109/L, absence of leukemia blast cells, normal marrow differential, and complete resolution of extramedullary disease. CRi is CR but platelets are \< 100x109/L or ANC is \<1x109/L. HI is described by the number of individual, positively affected cell lines without the use of growth factors and/or transfusions (lasting at least 4 weeks).

Incyte Corporation

M.D. Anderson Cancer Center

Phase I starting dose of Ruxolitinib 50 mg by mouth twice a day (BID) for 28 day cycle.

Ruxolitinib 50 mg BID

Ruxolitinib 100 mg BID

Ruxolitinib 200 mg BID

Total

Age, Continuous

Sex: Female, Male

United States

Region of Enrollment

Srdan Verstovsek, Professor, Leukemia

Number of Participants With Dose Limiting Toxicities (DLTs)

Phase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle.

Phase II - MTD reached in Phase I.

Ruxolitinib: Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle.

Phase II - MTD reached in Phase I.

Maximum Tolerated Dose (MTD) of Ruxolitinib

The study was stopped because of a lack of satisfactory clinical benefit, and did not go on to the Phase II portion. Therefore, data were not collected for this outcome measure.

Participants With a Response

Overall Study

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Precancerous Conditions

Part 1: Dose of ruxolitinib received will depend when patient joined study. The first group of patients receive the lowest dose of ruxolitinib. Starting dose level for Part 1, 5 mg by mouth twice a day for a 28 day cycle. The second group of patients receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of patients receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of patients take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond.

If patients enrolled in Part 2, they will receive ruxolitinib at the highest dose that was tolerated in Part 1.

Starting dose level for Part 1: 5 mg by mouth twice a day for a 28 day cycle.

Starting dose level for Part 2: Maximum tolerated dose from Part 1.

Guillermo Garcia-Manero, MD

The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with low or intermediate-1 risk MDS. The safety of this drug will also be studied, and whether it can help to control the disease.

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 4 groups of 3-6 participants will be enrolled in Part 1 of the study, and up to 7 participants will be enrolled in Part 2.

If you are enrolled in Part 1, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose of ruxolitinib. The second group of participants will receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of participants will receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of participants will take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond.

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

Study Drug Administration:

Each cycle is 28 days.

You will take ruxolitinib by mouth 2 times a day (about 12 hours apart), with or without food.

Study Visits:

One (1) time a week during Cycles 1 and 2 and then on Day 1 of Cycles 3 and beyond, blood (about 2-3 teaspoons) will be drawn for routine tests. On Day 28 of each cycle and Day 1 of Cycles 3 and beyond, this blood may also be used for cytogenetic testing if your doctor thinks it is needed.

On Day 28 of Cycles 1 and 2 and then every 3 cycles, you will have a bone marrow aspiration to check the status of the disease and for cytogenetic testing, if your doctor thinks it is needed.

On Day 28 of Cycle 1 and Day 1 of Cycles 3 and beyond, you will have a physical exam.

If the doctor thinks it is needed, urine will be collected on Day 1 of each cycle for routine tests.

Length of Dosing:

You may continue taking the study drug for up to 2 years. 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 after the end-of-dosing visit.

End-of-Dosing Visit:

After you are finished taking the study drug:

* You will have a physical exam.
* You will have a bone marrow aspiration to check the status of the disease.
* Blood (about 2-3 teaspoons) will be drawn for routine tests. This blood may also be used for cytogenetic testing if your doctor thinks it is needed.

Follow-up Visit:

About 30 days after the End-of-Dosing visit, a member of the study team will contact you by phone. You will be asked how you are feeling and about any side effects you may be experiencing. This phone call should take 10-15 minutes.

This is an investigational study. Ruxolitinib is FDA approved and commercially available for the treatment of myelofibrosis. Its use in this study is investigational. The study doctor can explain how ruxolitinib is designed to work.

Up to 31 participants will be enrolled in this study. All will take part at MD Anderson.

Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Phase I Study of Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Maximum tolerated dose (MTD) is the maximum dose at which \<33% of patients experience a dose limiting toxicity (DLT). Dose limiting toxicity defined as any grade 3 or higher non-hematologic toxicity.

Criteria for response follows the modified International Working Group (IWG) response criteria for altering natural history of MDS defined as: Complete Remission, Partial Remission, Stable Disease, Cytogenetic Response, Disease Progression.

Spots Global Cancer Trial Database for jakafi

Every month we try and update this database with for jakafi cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Phase l/II Study of Ruxolitinib for Acute Leukemia	NCT01251965	Leukemia	Ruxolitinib	14 Years -	M.D. Anderson Cancer Center
Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)	NCT01895842	Leukemia	Ruxolitinib	18 Years -	M.D. Anderson Cancer Center
Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)	NCT01895842	Leukemia	Ruxolitinib	18 Years -	M.D. Anderson Cancer Center

Spots Global Cancer Trial Database for jakafi

Every month we try and update this database with for jakafi cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for jakafi cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.