Azacitidine in Subjects with IPSS Lower-risk Myelodysplastic Syndromes
Research type
Research Study
Full title
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.
IRAS ID
114717
Contact name
Paresh Vyas
Sponsor organisation
Celgene Corporation
Eudract number
2012-002471-34
Clinicaltrials.gov Identifier
Research summary
MDS is a condition that affects the blood and bone marrow. Every day the bone marrow, your blood factory has to produce fresh new blood cells to allow you to live a healthy life. In MDS, blood cell production is abnormal and there is inefficient production of red blood cells, this leads to anaemia and this in turn causes fatigue. MDS can also cause a reduction in blood cells called white cells that predispose patients to infection. Finally, there is a reduction / can be a reduction in a blood cell type called platelets and this can predispose to bleeding and bruising. Azacitidine is a type of drug that changes how diseased cells in the blood and bone marrow grow and multiply. A liquid form of Azacitidine given by injections under the skin or intravenously, is approved for treatment of MDS in many countries. However, the oral form of Azacitidine has not been approved for treatment of MDS and is experimental in this study.The main purpose of this phase 3 study sponsored by Celgene Corporation is to see if participants with this type of MDS need fewer blood transfusions if they take oral Azacitidine. The safety and effectiveness of the drug will also be assessed and compared.Approximately 386 participants worldwide will take part in this study with approximately 25 participants being recruited in the UK. The total time the participant will be in the study could be up to 60 months.One half of the participants in this study will be assigned by chance into a 300 mg oral Azacitidine plus best supportive care study group and the other half will be assigned in a placebo (dummy drug) plus best supportive care group.This study involves procedures including: physical exam; vital signs; ECG; blood and urine samples; bone marrow samples; genetic tests; swabs; questionnaires.
REC name
East of Scotland Research Ethics Service REC 2
REC reference
13/ES/0005
Date of REC Opinion
19 Feb 2013
REC opinion
Further Information Favourable Opinion