A Phase 3, Multicenter, Randomized, Double- Blind Study To Compare The Efficacy And Safety Of Oral Azacitidine Plus Best Supportive Care Versus Placebo Plus Best Supportive Care In Subjects With Red Blood Cell Transfusion-Dependent Anemia And Thrombocytopenia Due To IPSS Lower-Risk Myelodysplastic Syndromes
ID Number 13-0956Principal Investigator(s)
Lewis R Silverman
Department(s) or Division(s)
Hematology and Medical Oncology
The main purpose of this study is to see if people with your type of MDS need fewer blood
transfusions if they take a drug called azacitidine by mouth (also called oral azacitidine).
The study sponsor and is working with your study doctor to find out the following information:
• What effect, good and/or bad, the study drug has on you and your type of MDS;
• what effect, good and/or bad, the study drug has on your quality of life (your general
• how much of the study drug is taken into the bloodstream, how long the study drug stays
in the body, and the connection between any effects and the study drug;
• if there are any genetic and chemical abnormalities present in your diseased cells in the
blood and bone marrow and whether these abnormalities, if seen, play a role in how your
disease responds or does not respond to the study drug.
Note: Testing of genetic material (also called deoxyribonucleic acid [DNA] and
ribonucleic acid [RNA]) is required for this study. Genetic tests will not tell if your
disease is inherited and will not involve members of your family. In this consent form,
you will have the chance to indicate whether you allow the testing of your genetic
material. If you do not allow this testing, you cannot participate in this study.
The study drug in this study is either oral azacitidine or placebo (sometimes called a sugar pill).
Placebo looks like the study drug but is not active drug.
Azacitidine belongs to a class of anti-cancer drugs known as DNA de-methylating agents. These
types of drugs change how diseased cells in the blood and bone marrow grow and multiply.
Vidaza®, a liquid form of azacitidine given by injections under the skin or intravenously, was
approved for treatment of MDS in US, EU and 30 other countries including Canada,
Switzerland, Australia and Japan. However, the oral form of azacitidine has not been approved
for treatment of MDS and is experimental (being tested) in this study. An experimental drug is
not approved by the United States Food and Drug Administration (FDA) for use except in
Recruiting Patients: Yes