Acute Leukemia

Clinical Trials

Below is a list of Acute Leukemia clinical trials from the clinical trials database at Mayo Clinic.

This list includes only trials about which Mayo researchers choose to publish information. Mayo Clinic may be conducting other trials which are not in this database. Mayo's clinical trials include experimental treatments, often unavailable elsewhere, which frequently lead to improved patient care for people worldwide. Patients should ask their doctor at Mayo about clinical trials appropriate for their situation.

A Phase 3 Study of Clofarabine and Cytarabine (ara-C) for Older Patients With Relapsed or Refractory (Resistant) Acute Myelogenous Leukemia (AML)
This study is being done to find out what effects (good and bad) clofarabine (also called Clolar) has on a patient and their acute myelogenous leaukemia (AML). Clofarabine is currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of pediatric patients with acute lymphoblastic leukemia (ALL) who have not responded or have relapsed after receiving two prior treatments. Early clinical studies using clofarabine in adult patients with AML were conducted. However, clofarabine is not approved by the FDA for the treatment of AML; it is still investigational in this disease. Cytarabine (also called ara-C) is an FDA approved chemotherapy that is given as a treatment for AML.
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Phase 3 Vaccine Study of Proteinase 3 PR1 Peptide Mixed with Montanide ISA-51 VG Adjuvant and Administered with GM-CSF in Elderly Patients with Acute Myelogenous Leukemia (AML) in First Complete Remission or Adults in Second Complete Remission: A Pivotal Study
Patients are being asked to take part in this research study because they have Acute Myelogenous Leukemia (AML) that is in complete remission. The goal of this research study is to see if a new vaccine (called PR1) can prolong the duration of their complete remission from AML and help patients live longer.


The PR1 vaccine consists of two components. The vaccine includes part of a protein that is made by the leukemia cells. This protein fragment is called a peptide. Another component of the vaccine is an oil-based liquid to help the body respond to the vaccine. The liquid is called an adjuvant. The name of the specific adjuvant in this study is Montanide ISA-51 VG. The vaccine may help fight leukemia by making a patient's normal white blood cells kill the leukemia cells. The vaccine is administered subcutaneously (under the skin, like an insulin shot) on a patient's thighs and arms.
Patients will also receive a drug called GM-CSF (granulocyte-macrophage colony stimulating factor) through a needle under the skin in the same area as the PR1 vaccine.
GM-CSF is a drug that makes blood cells grow and may also make the vaccine stronger.
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A Phase 3 Randomized Trial of Gemtuzumab Ozogamicin (Mylotarg) Combined with Conventional Chemotherapy for De Novo Acute Myeloid Leukemia (AML) in Children, Adolescents, and Young Adults
Acute myeloid leukemia (AML) is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone marrow. They may flood the bloodstream and invade vital organs such as the brain, testes, ovaries, or skin. These cancerous AML cells can sometimes form a solid tumor called a chloroma.

The standard treatment for this disease is to use a combination of cancer-fighting drugs called chemotherapy. Chemotherapy destroys the leukemia cells in the blood and bone marrow. In the first phase, called Induction 1, the medical team tries to remove all visible signs of leukemia and allow normal blood cells to be restored. This is called remission. The next phase of treatment is called Induction 2. Induction 2 is another round of chemotherapy to kill the few remaining leukemia cells that may have survived Induction 1. In the next phase of treatment, called Intensification 1, more chemotherapy is used to kill any remaining blast cells. Then the medical team does a Stem Cell Transplantation (SCT) or gives additional high doses of chemotherapy to try to keep the leukemia from coming back.

Nearly 500 children are diagnosed with AML every year, and half are cured with standard therapy. In other words, half of the children diagnosed with AML and treated as described above remain with no signs of cancer (remission) for five years. The overall goal of this study is to see if the research staff can increase this cure rate without causing more serious side effects of therapy. Side effects are unintended and unwanted results of treatment.

Researchers want to know if they can improve the cure rate for AML by adding a new chemotherapy drug, called gemtuzumab, to the standard chemotherapy treatments.
Gemtuzumab has been studied in adults with AML in combination with standard chemotherapy drugs. It has also been studied in small groups of pediatric patients. These studies have determined what dose of gemtuzumab can be given safely with other chemotherapy drugs.

Another goal of this study is to determine which children with AML need a stem cell transplant and which children do not. During intensification treatment, the study doctors will assign patients to either receive more chemotherapy, or a stem cell transplant. This is decided based on two factors: 1) the patient's risk of AML coming back, and 2) if they have a matched stem cell donor available.

The research staff will also be studying how well children with Down syndrome do on the standard therapy. Down syndrome patients may respond differently to chemotherapy drugs than patients without Down syndrome. This study will give study doctors information about how Down syndrome patients respond to standard therapy.

A secondary goal of the study is to understand the biology of AML better. These tests are optional and will be done only if the patient agrees. Study doctors want to test blood or bone marrow for certain genetic changes in leukemia cells. This would help them to learn more about AML and how to treat patients better. They also want to look for very small amounts of leukemia. This is called minimal residual disease (MRD). Researchers want to find out if measuring MRD can be used in the future to decide how great the risk of relapse is for a person.

In summary, the goals of this study are:
1. To see if adding a new drug called gemtuzumab to the current standard AML treatment will improve the cure rate without causing more serious side effects
2. To compare the outcomes of children who have higher risk disease and receive a stem cell transplant to children who do not have higher risk disease
3. To evaluate how well children with Down syndrome (who are over four years old) do when they receive the standard chemotherapy without gemtuzumab
4. To understand the biology of AML better with the optional biology tests

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A Pilot Study to Determine the Toxicity of the Addition of Rituximab to the Induction and Consolidation Phases and the Addition of Rasburicase to the Reduction Phase in Children with Newly Diagnosed Advanced B-Cell Leukemia/Lymphoma Treated with LMB/FAB Therapy
This study is being done to:
- Find out the effects, good and bad, of adding two new drugs, rituximab and rasburicase, to the standard chemotherapy drugs
- Find out the effects, good and bad, of rasburicase when given to see if it can lower the uric acid levels (salts found in patient's bloodstream)
- Compare the amount of cancer cells in the body before, during and after treatment
- Find out if there are proteins or genetic information (genes patients inherit from their parents) in the tumor cells that can tell researchers the best way to treat children with B-cell leukemia/lymphoma.

Non-Hodgkin lymphoma is a cancer of the lymph nodes (tissue throughout the body that filters disease germs from the blood). B-cell leukemia is cancer of the blood that develops in the bone marrow where blood cells are made. (ALL is a disease in which too many underdeveloped [not normal] infection-fighting white blood cells (blasts) crowd out the normal cells).

The first investigational drug, rituximab, is an anti-cancer drug that attaches to lymphoma cells and causes these cancer cells to die by stimulating the body's own immune system (the system that recognizes and attacks cells that are not normal body cells). Rituximab has been given safely along with standard chemotherapy to adult NHL patients.

However, rituximab has not yet been used in combination with standard chemotherapy to treat children.

The second drug given on this trial is called rasburicase. Rasburicase is a drug that lowers uric acid levels in the blood. Uric acid levels go up because of the death of the normal cells and tumor cells when the chemotherapy is given. Too much uric acid in the blood can cause the kidneys to fail. Researchers want to see if rasburicase can lower the uric acid levels, but they also need to find out what kind of side effects children may have when they take rasburicase. The standard drugs used to treat B-cell lymphoma are vincristine, prednisone and cyclophosphamide.
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AALL06N1, "A Study of Neurocognitive Function in Children Treated for Acute Lymphocytic Leukemia (ALL)"
Patients are being asked to take part in this research study because they have been diagnosed with acute lymphocytic leukemia (ALL) and have agreed to be treated on a COG treatment study. In this study, the researchers want to learn more about children who are treated on the COG studies AALL0232 and AALL0434. In both these treatment studies, patients (people on the study) are randomized (randomized means assigned randomly like the flip of a coin, but it is done by a computer so no one will know what treatment the patient will receive) between two different treatment arms that compare different ways to give the chemotherapy, methotrexate. Methotrexate is given in a two month long phase of treatment called Interim Maintenance.

In this study, the research staff want to closely evaluate side effects that might be due to methotrexate. Specifically, the researchers want to learn more about possible side effects that affect the nervous system. A recent, large study of adult survivors of childhood ALL found no differences between survivors who never received radiation treatments and the general population with respect to the likelihood of being married, having a job, and having health insurance.

However, there are children who have neurologic side effects during therapy, or who have learning disabilities after therapy has ended. This study is looking at whether the use of methotrexate is likely to cause those side effects that affect the nervous system. This study will also look at whether or not the possible side effects are different with the different ways methotrexate is given.


Why Is This Study Being Done?
The study is being done to determine the effects, if any, of methotrexate, on learning skills and memory. Information gathered from previous studies is incomplete. There is currently no way of predicting who is likely to develop nervous system side effects related to the use of methotrexate, regardless of the way that methotrexate is given. Side effects are unintended or unwanted results of treatment. In addition, there is no way of predicting whether the nervous system side effects will be minor or major.

This study will also determine: 1) Whether rare, sudden side effects of leukemia therapy, such as seizures, are associated with learning difficulties; 2) Whether certain genetic traits (qualities of a person?s DNA that make the person unique) are associated with the possibility of methotrexate side effects; 3) Whether changes in the amounts of a substance that is found in the patient?s body are associated with side effects of methotrexate, and; 4) Whether or not a new imaging study, similar to an MRI, will predict who is at risk for side effects related to methotrexate.

The overall goal of this study is to evaluate the potential toxicity associated with methotrexate in more detail.
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AAML0431, The Treatment of Down Syndrome Children with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) Under the Age of 4 Years
Parents/ Guardians are being asked to allow their child take part in this study because their child has Down syndrome (DS) and has recently been diagnosed with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS).

AML is a cancer of the bone marrow, the spongy tissue inside large bones where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood
cells called blasts. These blast cells crowd out the normal cells of the bone marrow. They may flood the bloodstream and invade vital organs such as the brain, testes, ovaries, or skin. These cancerous AML cells can sometimes form a solid tumor called a chloroma. Many patients have MDS before they get AML. MDS is a disease in which the body
makes fewer blood cells than usual. Bone marrow in MDS patients does not produce enough healthy blood cells. MDS can develop into leukemia.


What Is The Current Standard Of Treatment For This Disease?
The standard treatment for AML and MDS is to use a combination of cancer-fighting drugs called chemotherapy. Chemotherapy destroys the leukemia cells in the blood and bone marrow. The standard treatment regimen consists of two phases of therapy, called Induction and Intensification. In the Induction phase we try to remove all visible signs of leukemia and allow normal blood cells to be restored. This is called remission. Induction treatment is usually repeated for three cycles of therapy (each cycle 28 days). The next phase of treatment is called Intensification. Intensification chemotherapy is used to kill the few remaining leukemia cells that may have survived Induction. Intensification is usually three cycles of therapy, and includes high dose cytarabine (one of the chemotherapy drugs) in the last cycle. Patients with AML or MDS may also be treated with up to seven doses of cytarabine that is injected into the spinal fluid.

Why Is This Study Being Done?
Research has shown that children with DS are more likely to develop leukemia than children who do not have DS. However, they are also known to respond better to chemotherapy than children with AML who do not have DS.

The overall goal of this study is to see if we can increase the cure rate and decrease the side effects of therapy. Side effects are unintended and unwanted results of treatment. In this study, we will test the effects good and/or bad of changing the order of one of the chemotherapy treatments, high dose cytarabine (Ara-C). Subjects in this study will receive high dose cytarabine earlier in the treatment schedule than in past studies. Since DS patients do well on chemotherapy, study doctors want to see if it is possible to lower the side effects of chemotherapy without lowering the effectiveness of the treatment. Study doctors would like to know the effects good and/or bad of reducing the following chemotherapy treatments:

1) The number of treatments given in the spinal fluid (called ?intrathecal?)
2) The number of doses of daunorubicin, one of the chemotherapy drugs

A secondary goal of the study is to learn more about the biology of AML and MDS in DS patients. These tests are optional and will be done only if the parent/ guardian agree. Briefly, the biology studies will:
? Test for genetic changes in the leukemia cells, and genetic factors which might affect a patient?s likelihood of getting leukemia and outcome with treatment
? Look for very small amounts of cancer cells in the blood and bone marrow, called minimal residual disease (MRD). Researchers want to find out if measuring MRD can be used in the future to decide how great the risk of the cancer coming back is for a person and predict how a patient will do with treatment
? See what happens to high dose cytarabine (one of the chemotherapy drugs) in the body and how much of the drug remains active over an 8-hour period. These are called pharmacokinetic (PK) tests.
? Collect blood and bone marrow specimens and store them in a cell bank for future research into Down syndrome

In summary, the goals of this study are to:
1. See if changing the order of high dose cytarabine in the treatment plan has an affect on the cure rate for DS patients
2. See if lowering the number of treatments into the spinal fluid and the number of doses of daunorubicin will be as effective as standard treatment with fewer side effects
3. Understand the biology of AML and MDS better with the optional biology tests
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C0701a/501, An Open-Label Extended-Use Study of Oral CEP-701 in Patients with Hematologic and Non-Hematologic Malignancies who have Completed a Clinical Study of CEP-701
This study is being done to continue comparing what effects (good and bad) the study drug CEP-701 has on you and your Acute Myeloid Leukemia. The study that you previously participated in shows that you may be experiencing good effects from the treatment, so you can continue to receive treatment.
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Combination Chemotherapy (Methotrexate, Nelarabine) in Treating Young Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is a type of cancer that occurs in the bone marrow. It is a disease in which there is an uncontrolled growth of abnormal white blood cells, referred to as blasts. These abnormal cells crowd out the normal cells in the bone marrow. Sometimes these blasts cells can be found in the brain, spinal cord, and/or other organs of the body. It is common to enroll children and adolescents with cancer in a clinical trial that seeks to improve cancer treatment over time. Clinical trials include only people who choose to take part. You have a choice between a standard treatment for T-cell ALL and this clinical trial.

What Is The Current Standard Of Treatment For This Disease?
In the first stage, called Induction, the medical team tries to remove all visible signs of leukemia and allow normal blood cells to be restored (this is called remission). Six chemotherapy drugs are used during this stage of treatment. The leukemia treatment is the same for patients who choose to be on this study and those who do not.

The following is detailed information on the current standard of Induction therapy:

Standard Treatments and Procedures for Patients with T cell ALL

Central Line
For drugs to be given by vein, the doctor will likely recommend that the patient have a central venous line placed.

Methods for Giving Drugs
Various methods will be used to give drugs to patients.
- PO - Drug is given by tablet or liquid swallowed through the mouth.
- IV - Drug is given using a needle or tubing inserted into a vein. It can be given by IV push over several minutes or by infusion over minutes or hours.
- IM - Drug is given using a needle injected into the muscle.
- SQ - Drug is given by injecting a needle into the tissue just under the skin.
- IT - Drug used to treat the brain and spinal cord is given using a needle inserted into the fluid surrounding the spinal cord.

Most drugs used on this study will be given using a needle or tubing inserted into the vein (IV).

Therapy for patients on all treatment plans:
The purpose of Induction therapy is to kill as many of the leukemia cells as possible so the disease goes into remission.

Drugs/ How the drug will be given/ Days
Cytarabine IV or SQ Day 1
Vincristine IV Days 1, 8, 15, 22
Prednisone PO or IV Days 1 - 28
Daunorubicin IV Days 1, 8, 15, 22
PEG Asparaginase* IM Days 4 [or 5 or 6]
Methotrexate** IT Days 8, 29
Leucovorin (patients with Down syndrome ONLY) PO 48 and 60 hours following each IT MTX

*If patients develop an allergy to PEG asparaginase, six injections of a different form of asparaginase (called Crisantaspase) may be substituted for each dose of PEG asparaginase

**If patients have leukemia in the spinal fluid of the first spinal tap they may also get methotrexate on Days 15 and 22


Standard tests and procedures
The following tests and procedures are part of regular cancer care and may be done even if the patient does not join the study.
- Physical exams
- Frequent labs to monitor blood counts and blood chemistries
- Urine tests to measure how the kidneys are functioning
- Pregnancy test for females of childbearing age before treatment begins
- X-rays and scans to monitor the patient's response to treatment
- Tests to monitor heart and lung functioning
- Bone marrow aspirations to see if the leukemia is responding to treatment
- Spinal taps to check for leukemia cells in the spinal fluid and to give chemotherapy into the spinal fluid

Measurement of Early Response to Treatment
During Induction therapy, tests will be done on the patient's bone marrow samples to let the researchers know how well the patient is responding to treatment. Two types of tests will be done.
1. The patient's local hospital laboratory will perform standard tests to measure how much leukemia is still left.
2. The central Children's Oncology Group (COG) laboratory will use a specialized test to measure minimal residual disease. This test detects numbers of leukemia cells that are too small to be seen by human eyes.

Using the results of these tests, in combination with the patient's age and white blood cell count at diagnosis and whether or not the patient had leukemia in their spinal fluid or testicles, researchers will then be able to place the patient into one of four categories:

1. Low Risk: These patients are less than 10 years old and have a white blood cell count of less than 50,000 at diagnosis, without evidence of testicular disease or leukemia in the central nervous system. In addition, these patients have very few or no leukemia cells in the bone marrow tested in your local hospital by Day 8 or Day 15 and there is little minimal residual disease found in the bone marrow tested at the COG laboratory from Day 29. Low Risk patients will not take part in the study of the new drug, nelarabine, nor will they receive radiation therapy since they are expected to do well with standard treatment.
2. Intermediate Risk: This group included all of those patients who do not meet criteria or fit the definition for Low Risk, High Risk or patients who still have T cell leukemia at the end of Induction.
3. High Risk: Regardless of any other features or test results, these patients have 5 percent to 25 percent blasts visible under the microscope on Day 29 of Induction therapy OR more than 1 percent blasts detected by a special test for minimal residual disease (MRD).
4. Patients with T cell leukemia at the end of Induction: Regardless of any other features or tests results, these patients have more than 25 percent blasts visible under the microscope on Day 29 of Induction therapy.

The term "risk" refers to the chance of the cancer coming back after treatment. Once the researchers have measured the patient's early response to treatment, they will be able to find out which of these risk categories of T cell ALL the patient belongs to. This will help determine the amount of chemotherapy the patient will need for the second part of the treatment.
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Combination Chemotherapy and Radiation Therapy in Treating Patients With Acute Lymphoblastic Leukemia (ALL) That Has Relapsed in the Central Nervous System (CNS) or Testes
This study is being done to see if stronger chemotherapy (anti-cancer drugs) used with less radiation therapy can bring about a remission (make the cancer go away).
Patients are being asked to take part in this study because they have acute lymphoblastic leukemia (ALL). At least 18 months have passed since they were first diagnosed with ALL. The leukemia had gone away (been in remission) but now it has returned (relapsed). The leukemia returned in their central nervous system (brain and spinal cord) and the testes.
The research staff know that radiation therapy can cause long-term side effects (side effects are unintended physical reactions to the drugs that are unrelated to the reasons the drugs are being used). The researchers hope to find out if this study treatment will still make the same high rates of remission that are seen with standard treatment but cause less long-term side effects. In addition to these treatment aims, the research staff would like to use information collected on this study to answer some research questions that might help future patients. Patients can choose to be on this clinical trial without taking part in this piece of the research.

The research aims are as follows:
- See if tiny amounts of leukemia cells might be present in the bone marrow (the soft tissue in the hollow of flat bones of the body that makes new blood cells) at the time of relapse (study entry). Researchers want to know if the amount of these cells can tell the research staff how well a patient might respond to treatment.
- Look for changes in the genetic structure (DNA) of relapsed patients. The research staff want to see if genetic changes make a difference in how the patient responds to treatment and in the types of side effects the patient has while being treated.
- Look at the effects that the disease and treatment have on thinking, learning, and behavior. The research staff want to know if patients have fewer learning and other psychological problems if less CNS radiation therapy is given.
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Drug Therapy for the Treatment of Acute Myeloid Leukemia (AML)
This study is being done to find out if the study drug CEP-701 can improve the treatment of your Acute Myeloid Leukemia (AML). The study will also provide safety information of CEP 701 and the level of drug in your blood. If you achieve complete response (your disease does not progress or relapse), you may be able to continue taking the drug by taking part in another study.
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Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission after Primary Induction Failure or Patients over Age 60 in First Remission
This research study is being done to determine how well the investigational drug called farnesyl transferase inhibitor (FTI) R115777 can keep your cancer in remission (when the disease is less active) and to see what side effects (good and bad) the drug causes.

This research is being done because the average time of remission without treatment is 4 months. Even though you are currently in remission, there remains a great chance that your leukemia will come back despite the treatment that you have received. There is currently no effective additional treatment to keep your cancer in remission.
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Intensive Treatment for Intermediate-Risk Relapse of Childhood B-Precursor Acute Lymphoblastic Leukemia (ALL): A Randomized Trial of Vincristine Strategies
Patients have been treated in the past for a cancer of the blood cells called acute lymphoblastic leukemia (ALL). Patients are being asked to take part in this study because the leukemia has come back (relapsed) and is of intermediate risk. The term "risk" refers to the chance of the cancer coming back again after treatment for this relapse. The patient's leukemia is defined as "intermediate risk" because one of the following apply:
-The leukemia returned in their bone marrow (with or without leukemia in
other parts of their body) 3 years or more after they were first diagnosed
with ALL.
-The leukemia returned in the patient's central nervous system (spinal fluid) and/or the testes (but not in the bone marrow) less than 18 months after they were first diagnosed with ALL.

It is common to enroll children and adolescents with cancer in a clinical trial that seeks to
improve cancer treatment over time. Clinical trials include only people who choose to
take part. Many different chemotherapy treatment programs have been used for children with intermediate risk relapse of ALL, and there is no real standard treatment at this time. Patients have a choice between other treatments for ALL and this clinical trial.

What Is The Current Standard Of Treatment For This Disease?
There is no generally accepted standard treatment for intermediate risk relapse of ALL at this time. Combination chemotherapy has been the main treatment for children with intermediate risk relapsed ALL. Several studies have used various combinations and had similar outcomes in the number of children treated successfully.

The first month of treatment usually involves chemotherapy very similar to that given to treat ALL for the first time. Children with intermediate risk relapse have an excellent chance of going into a second remission (disappearance of all signs and symptoms of ALL).
However, the risk that the leukemia will come back after getting into remission is definitely higher than it was when the patient was first diagnosed with ALL. Because of this, the treatment that is given for relapse is more intensive (stronger) than that given to treat ALL for the first time. Like the first treatment for ALL, treatment for intermediate risk relapse has several phases. All phases of treatment are very important. The total length of chemotherapy treatment used for intermediate risk relapse varies from about 1.5 to 2.5 years in most cases.

Some doctors also recommend bone marrow (stem cell) transplants for some children and adolescents with intermediate risk relapse. This study will recommend bone marrow transplant for children with intermediate risk relapse that go into a second remission and have a matched family donor. About 20 to 30 percent of patients will have a close match. This use of bone marrow transplant is considered to be a valid treatment. Also, radiation therapy is sometimes used in relapsed ALL, especially if the relapse involves organs such as the brain or testes.

Why Is This Study Being Done?
Since there is no standard treatment regimen for children with intermediate risk relapse of ALL, the major goal of this study is to establish an effective therapy. The study treatment plan is based on an earlier plan (?POG 9412?) which showed a good effect in children who had a relapse of ALL in the brain. In this study, the treatment will be strengthened by giving higher doses of chemotherapy drugs more often. This study is to determine how effective this treatment is for all types of intermediate risk relapse.

Patients received vincristine during their first treatment for ALL, and it is part of treatments commonly used for relapsed ALL. The standard dose of vincristine is 1.5 milligrams/square meter (mg/m2)with a maximum dose of 2 mg. Higher doses of vincristine have been used to treat children with other forms of cancer and a recent Dutch trial has also used a higher dose to treat children with ALL. The researchers want to see if a higher dose of vincristine (2 mg/m2 with a maximum dose of 2.5 mg) can get rid of the cancer for as long as possible in more patients with relapsed ALL.


Researchers want to compare the effects, good and/or bad, of chemotherapy with a high dose of vincristine against chemotherapy with a standard dose of vincristine. The research staff want to find out which dose is better. In this study, patients will get either the high dose of vincristine or the standard dose of vincristine. Patients will not get both.

The goals of this study are:
1) To find out the effectiveness and side effects of the high dose combination chemotherapy treatment for all patients on this study.
2) To find out the effectiveness and side effects of higher dose vincristine compared to standard dose vincristine.

In addition to the treatment goals, researchers would like to use specimens collected on this study to answer some research questions that might benefit future patients. Patients can choose to be in this clinical trial without taking part in this research portion.

One of the biology research goals involves a test to measure minimal residual disease (MRD). This test measures numbers of leukemia cells that are too small to be counted using traditional methods. Researchers will use it to find out if leukemia is still present in the bone marrow. The research staff also want to study the genetic make-up of the cancer cells to try to learn about why people get cancer and if the genes in the cancer cells can predict how someone will respond to treatment.

Biology Research Goals (Optional Participation):
1) To find out if MRD levels can be used to tell how well a patient with relapsed ALL is responding to treatment. Researchers also want to find out if high MRD levels can be used to identify people at higher risk of another relapse.
2) To study genetic changes, and patterns within the genes of the leukemia cells. The research staff hope to learn more about disease resistance and find out if they can predict how well someone will respond to chemotherapy.
3) To find out if a) differences that occur naturally in genes (polymorphisms) might be part of the reason why some people develop leukemia and b) if those differences influence why some patients have certain side effects with treatment.

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Phase II Study with Azacitidine with or without (the Histone Deacetylase Inhibitor) MS-275 for the Treatment of Myelodysplastic Syndrome (MDS), Chronic Myelomonocytic Leukemia (CML) and Acute Myeloid Leukemia (AML)
The purpose of this study is to find out what effects, good or bad, one of two treatment programs has on a patient and their bone marrow disorder. One treatment program consists of a new dose and schedule of a drug called Azacitidine, which is approved for the treatment of myelodysplastic syndrome (MDS). The other treatment program includes the same dose and schedule of azacitidine in combination with a new drug known as MS-275. In this study patients will get either azacitidine alone, or azacitidine plus MS-275. Patients will not get both treatment
programs.

MS-275 is investigational and has not been approved by the U.S. Food and Drug Administration (FDA) for use in this cancer.
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Treatment Study for Patients Diagnosed with High Risk B-Precursor Acute Lymphoblastic Leukemia (ALL)
Leukemia is cancer of the blood. It starts in the bone marrow, which is the soft, spongy center of the long bones that makes the three major blood cells: white blood cells to fight infection; red blood cells that carry oxygen; and platelets that help blood clot and stop bleeding. Acute lymphoblastic leukemia (ALL) is a disease in which too many underdeveloped (not normal) infection-fighting white blood cells called "blasts", are found in the blood and bone marrow. These blasts crowd out the normal cells and can be found in the brain, spinal cord, and/or other organs of the body.

ALL is said to be "high risk" because patients have a white blood cell count of over 50,000 and/or they are ten years of age or older. Patients are also classified as "high risk' if any of the following applies:
- They are a male and have leukemia in their testes, regardless of their age and/or white blood cell count
- Patient has gotten treatment with steroid medicines, such as prednisone or decadron, in the month before they were diagnosed with leukemia

This study is being done to see if we can increase the cure rate of high risk ALL without increasing the side effects (unintended and unwanted results) of treatment.

The rate of survival keeps getting better for children and adolescents with high risk ALL. However, leukemia in the central nervous system (CNS) (brain and spinal cord) has become an increasing cause of treatment failure. This study will use an established chemotherapy regimen that has been very effective for treating children and adolescents with high risk ALL and will test if two different changes to this treatment can cure more patients.

Goal #1: To find out if using dexamethasone instead of prednisone during the first month of treatment can improve survival rates without causing more bad side effects. Prednisone and dexamethasone are closely related medicines called "steroids." In previous studies, steroids have been shown to be very effective against ALL, especially when given early in treatment. The standard treatment in high risk ALL is to give prednisone every day for 28 days during the first month of treatment.

When dexamethasone was used for all 28 days of the first month of treatment in many recent U.S. trials for children and adolescents with high risk ALL, there was a higher risk of infection. This included major life-threatening infections.

Because of this, the study will test two things:
- If 14 days of dexamethasone, instead of 28, can be taken without a higher number of bad side effects, and
- If 14 days of dexamethasone will be better than 28 days of prednisone in lowering the number of leukemia cells during the first month of treatment.

Goal#2: To find out if using high doses of methotrexate instead of using lower doses of
methotrexate on a schedule that gradually increases the dose, will keep the cancer from coming back without having more bad side effects.

Methotrexate is a cancer-fighting drug that is very important in the treatment of leukemia.
There are two different ways to give methotrexate during the interim maintenance stage of treatment. We know that both of these ways of giving methotrexate have been very effective in treating ALL, but they have never been directly compared to one another in children and adolescents with high risk ALL.

In"high dose" methotrexate treatment, the same dose of methotrexate is given a total of four times (every two weeks over a nine-week period).

In Capizzi methotrexate, methotrexate is given a total of five times (every 10 days over an eight-week period). Instead of being the same dose (as in high dose methotrexate treatment), Capizzi methotrexate is given at a lower dose the first time, and the amount of the dose slowly increases with each of the later doses. Capizzi methotrexate also includes treatment of another medicine, call PEG-asparaginase, which is given twice during this eight-week stage of treatment.

In addition to the goals related to the changes in treatment, there is an additional research
goal.
Goal#3: To see how quickly patients are helped by the induction phase (reach disease remission) and how well they do after treatment, as measured by the presence or absence of very small numbers of leukemia cells in the bone marrow after the first month of treatment.
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