IRB Number:

07-000229

Trial Status:

Open for Enrollment

Phase: II

Why is this study being done?

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).

Who is Eligible to Participate in the Study?

-Age 18 or older
-Diagnosed with Myelodysplastic Syndrome (MDS)
-No more than 2 prior therapies for MDS

What is Involved With this Study?

Before starting the study, patients will have the following:
-Medical history and physical exam
-Blood tests
-ECG (an electrocardiogram), which is a painless test involving an electrical measurement of the heart

Patients may also have other tests that their doctor thinks they need.

Patients will be given PXD101 in the vein once a day for the first five days of each 21 day cycle. They will keep getting treatment for four cycles of therapy, as long as their cancer does not get worse and they are not having bad side effects. After four cycles the patient may stay on treatment for another four cycles if their cancer is responding.

At the start of each new cycle, patients will have an office visit and a physical exam. (Each cycle lasts 21 days.) An ECG will be done on the fifth treatment day of each cycle. These tests are to find out the effects the drug is having on the patient. The patient's blood pressure should be checked each week during the first month of the treatment. The doctors will follow the effect of this treatment on the patient's Myelodysplastic Syndrome (MDS) by drawing blood each week during the first cycle of their treatment and then before every cycle after that, to check their blood counts. The patient will be asked to review with their doctor all their current prescription and over the counter medicines as well as any herbal or alternative therapies they may be taking, and any that are started while participating in this trial.

Patients will have a bone marrow biopsy done before starting treatment to assess the state of their MDS. Also, a bone marrow biopsy will be performed after every two cycles to assess their response to treatment and to look for abnormal cells in the bone marrow. The bone marrow biopsies are mandatory in order to assess the patient's response to the study treatment. These biopsies are part of a patient's regular patient care, and should be covered by their insurance company.

The drug used in this study is considered investigational, which means it has either not been approved by the U.S. Food and Drug Administration (FDA) for routine clinical use or for the use described in this study. However the FDA has allowed the use of this drug in this research study.

How long will the Study run?

Patients will get treatment with the study drug for up to eight cycles (total 24 weeks), or until it is no longer helpful, or if they or their doctor thinks that the side effects of the treatment are too serious. The patient will be followed for up to three years after starting the drugs so that the researchers can watch their health status.

Sponsor(s): Mayo Clinic

Study Activation/Registration Date: 10/05/2007

IRB Review and Approval Date: 01/25/2007

Study Type: Treatment

Projected Accrual: The plan is to have up to 55 patients participate from multiple medical centers.

Costs of Study:There may be standard patient care costs related to participating in a cancer research study.

Principal Investigator: David P. Steensma, MD

Who can I Contact for Additional Information on this Trial?

Complete Request Form for Cancer Clinical Trial Information at Mayo Clinic or
For more information about this study or other clinical trial options, please contact our Mayo Clinic Cancer Center Clinical Trials Referral Office.

• Patients living within the United States, call 507-538-7623, or e-mail cancerclinicaltrials@mayo.edu


• Patients living outside the United States may call our International Office at 507-284-8884, or e-mail intl.mcr@mayo.edu.

What is/are the Locations of this Clinical Trial?

• Rochester, MN

Last updated: 10/21/2008