5-azacitidine and Thalidomide for patients with Myelodysplastic Syndromes to assess safety and efficacy (MDS03)

Australasian Leukaemia and Lymphoma Group (ALLG)

Dataset description

Dataset includes Data for 80 patients with newly diagnosed MDS, utilising treatment with a combination of 5-azacitidine and Thalidomide Dataset includes demographic data, diagnostic, treatment data, outcome data Eg transfusion requirements, QoL
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Chemotherapy |

Source Study




Phase 1 / Phase 2


Commercial sector/Industry,Pharmion Australia

Scientific enquiries

Dr Melita Kenealy

Brief Summary

5-azacitidine, a demethylating agent, has been approved for use in USA for treatment of Myelodysplastic Syndromes (MDS), with overall response rates of approximately 48%, a delay to progression to acute leukaemia or death and an improvement in quality of life. Thalidomide has also shown some activity as a single agent in MDS though with poor tolerance at doses above 100mg/day. Neither of these agents is currently routinely available in Australia for MDS. This trial aims to show the safety and to ....
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Key Inclusion Criteria

1. Subjects with myelodysplastic syndrome (MDS), either de novo or treatment related 2. patients with refractory anaemia or refractory anaemia with ringed sideroblasts to meet one of the following additional criteria of marrow dysfunction:a. transfusion dependent or symptomatic anemia up to 3 monthsb. clinically significant thrombocytopenia; either significant bleeding, platelet transfusion dependency or thrombocytopenia with at least two counts less than or equal to 50x109/L at least 1 month ap ....
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Key Exclusion Criteria

1. bone marrow blast count 30% or more2. Grade 3-4 peripheral neuropathy3. prior stem cell transplantation4. prior treatment with thalidomide or its analogues within 30 days of commencing treatment on trial5. any prior treatment with 5-azacitidine, decitabine or any known demethylating agent6. treatment with Granulocyte colony stimulating factor in the 21 days prior to Day 1, androgenic hormones in 14 days prior to Day 1 or any investigational agent in the 30 days prior to Day 17. any serious me ....
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Can healthy volunteers participate?




Sample Size    80

Min. age    18 Years

Max. age    -2147483648 No limit

Sex    Both males and females

Condition category    Myelodysplastic Syndromes

Condition code    Blood


Intervention code Treatment: Drugs

All participants will receive both 5-azacitidine and thalidomide: 1. 5-azacitidine subcutaneous injection 75mg/m2/d for 7 days every 28 days for up to 24 cycles 2. Thalidomide orally commencing at 50mg/day, increasing to a maximum 100mg/day continuous treatment for up to 12 months


Control group Uncontrolled



Outcome: Haematologic and Non-haematologic toxicity of the combination of thalidomide and 5-azacitidine.
Timepoint: Toxicities are measured as they occur throughout the study period.

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