Phase 2 study of CC-486 alone & combined with Durvalumab in MDS pts
Research type
Research Study
Full title
A phase 2, international, multicentre, randomised, open-label, parallel group study to evaluate the efficacy and safety of CC-486 (oral Azacitidine) alone and in combination with Durvalumab (MEDI4736) in subjects with myelodysplastic syndromes who fail to achieve an objective response to treatment with Azacitidine for injection or Decitabine
IRAS ID
147355
Contact name
Elspeth Payne
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2014-002675-29
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 0 months, 31 days
Research summary
Summary of Research
Myelodysplastic syndrome (MDS) is a cancer of the blood that results in abnormal production of one or all of the blood cell types. These include red blood cells (that carry oxygen), white blood cells (that fight infection), and platelets (that help blood clot) which may be abnormal. Over time MDS may transform to leukaemia. Because of the potential to transform to leukaemia, most patients with MDS will be treated with hypomethylating agents (HMA) when there is evidence this is imminent.
This study is sponsored by Celgene Corporation, and will investigate the safety and effectiveness of two drugs, CC-486 either by itself, or in combination with durvalumab (MEDI4736). This study will enroll patients with MDS who have been treated with an HMA given by injection, but who did not respond.
CC-486 (oral azacitidine) is an HMA, but it is a pill and may work differently. CC-486 modifies the DNA that directs the cells to grow normally. By modifying the DNA, the MDS cells die or mature normally. Different dosages and schedules of CC-486 may result in DNA modification that have more beneficial effects than injectable HMA.
Durvalumab is an antibody that blocks a protein called programmed cell death ligand-1 (PD-L1). Signals from PD-L1 help cancer cells avoid detection by the immune system. By blocking PD-L1, durvalumab may allow the immune system to eliminate or control abnormal MDS cells. HMAs may also boost the immune response; therefore combining CC-486 with durvalumab might have a stronger overall effect.
This study has 4 parts: Screening, Safety Run-in or Randomised Treatment and Follow-up. Study procedures include physical examinations, vital signs, electrocardiogram, and the collection of bone marrow, blood, saliva and urine. Approximately 194 participants worldwide will take part. A patient could be in the study for 3 years.
REC name
London - Central Research Ethics Committee
REC reference
16/LO/0675
Date of REC Opinion
31 May 2016
REC opinion
Further Information Favourable Opinion