Phase 1/2 study of Azacitabine in subjects with AML / MDS after HSCT
Research type
Research Study
Full title
A PHASE 1/2, DOSE AND SCHEDULE FINDING STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PRELIMINARY EFFICACY OF ORAL AZACITIDINE (CC-486) IN SUBJECTS WITH ACUTE MYELOID LEUKEMIA OR MYELODYSPLASTIC SYNDROMES AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION
IRAS ID
127205
Contact name
Charles Craddock
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2012-005805-36
Research summary
MDS is a condition affecting the blood and bone marrow. In MDS, blood cell production is abnormal and there is inefficient production of red blood cells which leads to anaemia and fatigue. MDS can also cause a reduction in white blood cells that predispose patients to infection, and there can be a reduction in platelets which can predispose to bleeding and bruising. In some cases MDS can progress to AML in which increased production of leukaemia cells ‘spill over’ into the blood stream and circulate in the body, leading to an increased risk of infection as well as anaemia, bruising and bleeding.
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 injection is approved for treatment of MDS and a subset of AML in many countries. However, the oral form of azacitidine has not been approved and is experimental in this study.
The main purpose of this study sponsored by Celgene Corporation is to find the maximum tolerated dose of oral azacitidine in AML or MDS after Allogeneic Hematopoietic Stem Cell Transplantation. The safety and effectiveness of the drug will also be assessed.
Approximately 30 participants in the US and Europe will take part in this study. The total study duration will be approximately 30 months.
Participants will receive oral azacitidine at 200 or 300mg once daily for the first 7 or 14 days of each 28-day cycle. Dose and schedule received will be determined by the study doctor and will depend on what stage of the study the participant is enrolled.
This study involves: physical exams, vital signs, chest x-ray, ECGs, blood and urine samples, bone marrow samples and genetic tests.
REC name
North West - Haydock Research Ethics Committee
REC reference
13/NW/0347
Date of REC Opinion
10 Jun 2013
REC opinion
Further Information Favourable Opinion