A Randomised Phase II study comparing the efficacy of 5azacitidine alone versus combination therapy with lenalidomide and 5azacitidine in patients with higher risk myelodysplastic syndromes (MDS) and low marrow blast count acute myeloid leukaemia (AML). (MDS04)

Australasian Leukaemia and Lymphoma Group (ALLG)

Dataset description

Dataset includes: Data for 160 patients with newly diagnosed MDS. Patients were randomised to receive lenalidomide and 5azacitidine or 5azacitidine alone Demographic data, diagnostic data, treatment data, outcome data and measures of supportive care during treatment, Eg transfusion requirements, QoL
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Chemotherapy |

Source Study




Phase 2


Commercial sector/Industry,Celgene Pty Ltd

Scientific enquiries

Dr Dr Melita Kenealy

Brief Summary

Lenalidomide and Azacitidine each have clear evidence of efficacy in MDS, and have shown activity in AML. However, not all patients respond so better regimens, including combinations, are required. MDS and AML are heterogeneous diseases, and lenalidomide and azacitidine may target different cellular populations and induce differing clinical responses. Combinations of immunomodulatory drugs and azacitidine have been explored and shown to be feasible. This is an open label, multi-centre, randomise ....
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Key Inclusion Criteria

Disease diagnosis of either MDS (by World Health Organisation criteria, those with refractory anaemia and Refractory Anaemia with Ringed Sideroblasts to also have at least one clinically significant cytopenia), nonproliferative Chronic Myelomonocytic Leukaemia (CMML) or low marrow blast count AML; Eastern Cooperative Oncology Group (ECOG) 2 or less with life expectancy at least 3 months, adequate contraception and adequate renal and hepatic function, written informed consent.

Key Exclusion Criteria

Prior chemotherapy for MDS or AML except low dose cytarabine or hydroxyurea, any prior demethylating agent or immunomodulatory drug (including thalidomide or lenalidomide), prior diagnosis of cancer except with low risk of recurrence, significant renal, hepatic, cardiac or respiratory disease or severe active infection.

Can healthy volunteers participate?




Sample Size    160

Min. age    18 Years

Max. age    0 No limit

Sex    Both males and females

Condition category    Low marrow blast count acute myeloid leukaemia (AML) , Myelodysplastic syndromes (MDS)

Condition code    Blood , Cancer


Intervention code Treatment: Drugs

combination 5azacitidine (subcutaneous administration) and oral lenalidomide. All patients begin 5azacitidine 75mg/m2/day x 7 days (75mg/m2/day on days 1-5 and days 8-9) of a 28 day cycle. At Cycle 3, patients are randomised to commence lenalidomide (at 10mg/day x 21 days of a 28 day cycle) in combination with 5 azacitidine (75mg/m2/day x 5 days (days 1-5) of a 28 day cycle) until primary endpoint at 12 months after commencing treatment. Following this, all patients may continue 5azacitidine alo ....
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Control group Active

5azacitidine alone (subcutaneous administration; 75mg/m2/day on days 1-5 and days 8-9 of each 28 day cycle) for 12 months until primary endpoint, then all patients may continue 5azacitidine alone until progression or toxicity.


Outcome: To demonstrate improved efficacy with the combination of lenalidomide and 5azacitidine compared to 5azacitidine alone in patients with MDS and low marrow blast count AML, with acceptable toxicity of the combination. Outcome will be assessed by measures of disease response as defined by International Working Group (IWG) criteria (2006) and determined by peripheral blood counts, transfusions, bone marrow morphology and cytogenetics.
Timepoint: 12 months from start of ....

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Study Protocol: Study protocol
Data Dictionary: Not Available

Will individual participant data (IPD) for this trial be available?


What data in particular will be shared?

De-identified IPD data, for all data collected during the trial

When will data be available?

Data available 3 months following publication, for an indefinite period

Available to whom?

Data are potentially available to: • Researchers from not-for-profit organisations • Commercial organisations • Other Based in: • Any location Further information: All data requests will be considered by the primary sponsor on a case by case basis. Requests must include a methodologically sound proposal. Specific conditions of use may apply and will be specified in a data sharing agreement (or similar) that the requester must agree to before access is granted.

Available for what types of analyses?

Any type of analysis Assessed on a case-by-case basis

Source study information is derived from the Australian New Zealand Clinical Trials Registry (ANZCTR). For more information on the ANZCTR, please see