Myelodysplastic Syndromes

Clinical Trials

Below is a list of Myelodysplastic Syndromes 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 I and Pharmacological Clinical Trial of 17-Allylamino-17-demethoxygeldanamycin (17-AAG) and Cytarabine (Ara-C) in Refractory (Resistent) Leukemia and Myelodysplastic Syndrome (MDS)
- Find the highest dose of the cancer drugs called 17-AAG (17-Allylamino-17-
demethoxygeldanamycin) and cytarabine that can be given in a 60-day schedule to patients with advanced cancer without causing bad side effects
- Learn about the side effects of 17-AAG and cytarabine when given together
- Learn how the body handles or processes the drugs and learn the other effects the drugs have on the body using blood and bone marrow samples

17-AAG is an investigational drug that has not been approved by the U.S. Food and Drug
Administration (FDA), or any other regulatory agency, for commercial use but is approved by the FDA for use in this research study. Cytarabine has been approved by the FDA for the treatment of acute myelogenous leukemia. This study will provide information that will allow the investigators to further study if the combination is more effective than each drug given alone.
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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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AMG 531 for Myelodysplastic Syndrome (MDS) Patients Receiving Lenalidomide (Revlimid)
The purpose of this study is to identify a safe dose of AMG 531 that is best for treatment of low platelet count (thrombocytopenia) in subjects with low to intermediate-1 risk MDS who are being treated with Lenalidomide (Revlimid).
AMG 531 is considered investigational, which means that it has not been approved by the U.S. Food and Drug Administration. In this study, two or more dose levels of AMG 531 will be tested against placebo (a sugar or salt solution that is inactive and does not contain medicine). Because it is unknown whether treatment with AMG 531 is better than no treatment from thrombocytopenia, some participants will be assigned to receive a placebo.
AMG 531 is the investigational product being given in this study. AMG 531 is made of a protein that acts like a special protein in a person's body called thrombopoietin (TPO). TPO binds to the cells in a person's body that make platelets. This binding of TPO helps these cells to make more platelets. AMG 531 can also bind to these cells and help these cells to make more platelets.
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Comparison of Peripheral Blood Stem Cell Transplantation with Bone Marrow Transplantation for the Treatment of Serious Hematological Malignancies
This research study will compare the outcomes of study participants who have had peripheral blood stem cell transplantation from unrelated donors with those who have had bone marrow transplant from unrelated donors for the treatment of serious hematological malignancies.

Two types of stem cell transplantation have been used to treat patients with certain types of serious diseases. Stem cells can be obtained from bone marrow or from circulating blood (peripheral blood stem cells). Participants may be treated with a transplant of either bone marrow or peripheral blood stem cells (PBSC) from unrelated donors.

Both of these types of transplant have been successful for the treatment of leukemia and myelodysplasia. The goal of this research study is to see if there are better results using a bone marrow transplant or a PBSC transplant from unrelated donors.
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E1902, A Phase II Study of Reduced Intensity Allogeneic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes (MDS)
This study is being done to look at the effectiveness of an allogeneic transplant (a transplant where stem cells collected comes from the bone marrow or blood of another
person) using a less intense treatment regimen (a regimen that is thought to have less side effects) than a standard allogeneic transplant.

Patients are being asked to take part in this study because they have myelodysplastic syndrome and their doctors feel that they may not meet the requirements for a standard transplant either because age or other co-existent medical conditions that make the standard procedure too dangerous.

The goal of this study is to look at the effectiveness of an allogeneic transplant regimen that is thought to have less side effects. In a standard allogeneic transplant, high doses of chemotherapy and/or radiation therapy are used to kill bone marrow and myelodysplastic cells. Then new stem cells from blood or bone marrow from a different person are given, usually a family member (donor). This chemotherapy and radiation can cause many side effects in addition to destroying bone marrow. In this transplant regimen, patients will start with a process called photophoresis followed by chemotherapy and radiation at lower doses, in the hope that the treatment will cause less side effects than the high dose
treatment. After this treatment patients will get stem cells obtained from the blood (or bone marrow) of the donor. This treatment uses both chemotherapy and radiation in addition to the ability of the donor cells to destroy remaining abnormal cells in the recipient. This study involves research. To lower the patient's reaction to the donor cells, the patient's immune system will be suppressed using a process called photophoresis and a continuous infusion of pentostatin before radiation therapy and the allogeneic (donor) transplant. The researchers will look at the outcome of the transplant procedure with respect to side effects, safety, and effectiveness in treating the patient's disease. The research staff will also be looking at the patient's blood to see how much blood has been replaced by the donor's cells.
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Phase II Study of the Histone Deacetylase Inhibitor PXD101 for the Treatment of Myelodysplastic Syndrome (MDS)
Patients are being asked to take part in this research study because they have Myelodysplastic Syndrome (MDS). This study is being done to learn what effects (good and bad) PXD101 has on a patient and their Myelodysplastic Syndrome. Early studies of PXD101 and other drugs like it suggest that PXD101 can correct the abnormal expression of genes in cancer cells. This study will test whether PXD101 can improve blood counts and reduce bone marrow abnormalities in patients with myelodysplastic syndrome. Also, progression to acute leukemia, survival, and toxicity will be monitored in patients on this study. PXD101 is an experimental agent not yet approved by the U.S. Food and Drug Administration (FDA).
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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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