Oral Azacitidine in Subjects with AML in Complete Remission
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Subjects with Acute Myeloid Leukaemia in Complete Remission.
IRAS ID
123836
Contact name
Michael Dennis
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2012-003457-28
Clinicaltrials.gov Identifier
Research summary
Acute Myeloid Leukaemia (AML) is a disease that affects bone marrow (BM), the soft inner part of the bones, where new blood cells are made. Those cells would normally develop into different types of mature blood cells. In AML, there is an abnormal production of BM cells (immature leukaemic cells) which replace the normal blood-forming cells of the BM, accumulate and eventually spread into the blood stream. As a result, patients do not have enough normal red blood cells, white blood cells, and platelets. Thus, leading to increased risks of infection, anaemia, bruising and bleeding.
Azacitidine is a drug that changes how diseased cells in the blood and BM grow and multiply. Azacitidine, given by injection under the skin (subcutaneously) is approved for treatment of some forms of AML in many countries. Oral Azacitidine is not approved and is experimental in this study.
The main purpose of this randomised phase 3 study sponsored by Celgene Corporation, is to see if after standard medical treatment for AML patients who achieved complete remission (defined as having no evidence of disease) can benefit from oral Azacitidine as “maintenance therapy”, in continuing the response they had with their last AML treatment, reducing the risk for relapse.
Patients will be randomised (by chance) to one of the two treatment groups 1) oral azacitidine (300mg) plus best supportive care (BSC) and 2) placebo (dummy drug) plus BSC. All participants involved, undergo to study procedures including: physical exam; Electrocardiogram; blood and BM samples; genetic tests; and questionnaires.
Approximately 460 patients will take part worldwide, with about 30 from UK. Enrollment of all patients into the study is expected to take 30 months. The total time a participant will be in the study could be up to 60 months depending on when they enroll and whether the study drug is helping them or not.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
13/NW/0501
Date of REC Opinion
7 Aug 2013
REC opinion
Further Information Favourable Opinion