MEDI4736-MDS-001_Phase II_Durvalumab_MDS/AML
Research type
Research Study
Full title
A Randomized Multicenter, Open-label, Phase 2 Study Evaluating the Efficacy and Safety of Azacitidine Subcutaneous in Combination With Durvalumab (MEDI4736) in Previously Untreated Subjects with Higher-Risk Myelodysplastic Syndromes (MDS) or in Elderly (≥ 65 years) Acute Myeloid Leukemia (AML) Subjects Not Eligible for Hematopoietic Stem Cell Transplantation (HSCT)
IRAS ID
196245
Contact name
Charles Craddock
Contact email
Sponsor organisation
Celgene International II Sàrl
Eudract number
2015-003596-30
Clinicaltrials.gov Identifier
HAEM 5331, NIHR Clinical Research Network reference number
Duration of Study in the UK
2 years, 3 months, 24 days
Research summary
This is a randomised, multicenter, open-label, Phase 2 study evaluating the efficacy and safety of subcutaneous azacitidine in combination with durvalumab in two separate cohorts.
Cohort 1: patients with previously untreated MDS intermediate risk, high and very high risk, who are not eligible for Hematopoietic Stem Cell Transplantation (HSCT).
Cohort 2: patients with previously untreated AML who are elderly (≥ 65 years) and not eligible for HSCT, with intermediate or poor cytogenetic risk.
MDS encompasses a number of hematopoietic stem cell disorders. Anaemia is the most frequent manifestation of MDS, often progressing to red blood cell transfusion dependence. The overall survival in higher-risk MDS (24.5 months) is still insufficient. Approximately 35% to 40% of patients with MDS will develop AML.
AML is the most frequent form of leukaemia, accounting for 25% of all leukaemia’s in adults in the Western world. AML is a life-threatening disease primarily affecting older adults (>65), with a 5-year survival rate of 19%. The prognosis for elderly patients is far less favourable; only 5% of patients achieving a 5-year survival compared to 30% in patients aged 30 to 64 years.
Elderly patients not eligible for HSCT have a very poor prognosis with an overall survival of 6.5 months with conventional care regimens (CCR) and 10.5 months with Azacitidine.The purpose of the study is to evaluate the efficacy of subcutaneous azacitidine in combination with durvalumab as compared with subcutaneous azacitidine alone.
Approximately 182 eligible patients will be included in this study. Eligible patients diagnosed with either AML or MDS will be assigned to either the AML or MDS cohort. Approximately 72 patients will be included in the MDS cohort and 110 patients in the AML cohort.
The total duration of the study is expected at approximately 27 months, from first patient enrolled until last patient last visit.
REC name
South Central - Oxford C Research Ethics Committee
REC reference
16/SC/0068
Date of REC Opinion
23 May 2016
REC opinion
Further Information Favourable Opinion