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Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase I/II trial to determine safety & efficacy of combination therapy with 5-azacitidine (Vidaza) and Thalidomide in patients with Myelodysplastic Syndromes (MDS)
Scientific title
5-azacitidine and Thalidomide for patients with Myelodysplastic Syndromes to assess safety and efficacy
Secondary ID [1] 370 0
Australasian Leukaemia and Lymphoma Group (ALLG): ALLG MDS3
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 1824 0
Condition category
Condition code
Blood 1916 1916 0 0
Other blood disorders

Study type
Description of intervention(s) / exposure
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
Intervention code [1] 1765 0
Treatment: Drugs
Comparator / control treatment
Control group

Primary outcome [1] 2727 0
Haematologic and Non-haematologic toxicity of the combination of thalidomide and 5-azacitidine.
Timepoint [1] 2727 0
Toxicities are measured as they occur throughout the study period.
Secondary outcome [1] 4601 0
Complete response, partial response, haematologic improvement, time to relapse, disease progression, leukaemic transformation or death, change in Quality Of Life parameters, measures of methylation status of various genes, apoptosis and immune modulation.
Timepoint [1] 4601 0
Complete response, partial response, haematologic improvement, Cytogenic response and time to relapse are all measured at Visit 1 of Cycles 3-24, using the modified International Working Group (IWG) criteria.
Secondary outcome [2] 4602 0
Leukaemic transformation or death
Timepoint [2] 4602 0
Is recorded as it occurs throughout the study period.
Secondary outcome [3] 4603 0
Change in Quality Of Life parameters using the European Organisation for Research and Treatment of Cancer (EORTC QLC-C30) and Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaires.
Timepoint [3] 4603 0
Is measured at Screening, Cycle 5 Visit 1 and Cycle 13 Visit 1. Measures of methylation status of various genes, apoptosis and immune modulation, are all measured at Screening, Cycle 2 Visit 4, Cycle 4 Visit 4, Cycle 8 Visit 3, Cycle 12 Visit 3, Cycle 16 Visit 3, Cycle 20 Visit 3, and Cycle 24 Visit 3.

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 apartc. significant neutropenia with absolute neutrophil count = 1.0 x 109/L on at least 2 occasions 1 month apart (those patients with refractory anaemia with excess blasts, refractory anaemia with excess blasts in transformation and chronic myelomonocytic leukaemia are not required to meet the above additional criteria). 4. life expectancy 3 months or over5. Eastern Cooperative Oncology Group performance status 0-26. adequate hepatic function as defined by bilirubin = 1.5 x the upper limit of normal and aspartate aminotransferase & alanine aminotransferase = 2 x upper limits of normal 7. adequate renal function 8. provision of written informed consent prior to registration9. males with a female partner of childbearing potential must agree to use at least 2 effective contraceptive methods throughout the study and for 30 days following the date of last dose 10. women of childbearing potential may participate providing they agree to use at least 2 effective contraceptive methods throughout the study and for 30 days following completion, and have a negative serum pregnancy test within 21 days prior to commencement of study treatment.
Minimum age
18 Years
Maximum age
No limit
Both males and females
Can healthy volunteers participate?
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 medical condition which the investigator feels may interfere with the procedures or evaluations of the study8. history of other malignancy within 5 years, except if local disease completely excised with a high probability of cure 9. hepatic tumour or advanced liver disease10. significant cardiac or respiratory disease 11. known active viral infection with human immunodeficiency virus or viral hepatitis B12. known or suspected hypersensitivity to 5-azacitidine, mannitol or thalidomide13. pregnant or breastfeeding females 14. patients unwilling or unable to comply with study protocol15. current participation in another therapeutic clinical trial.

Study design
Purpose of the study
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?

Intervention assignment
Single group
Other design features
Phase 1 / Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 2060 0
Commercial sector/Industry
Name [1] 2060 0
Pharmion Australia
Address [1] 2060 0
Country [1] 2060 0
Funding source category [2] 2061 0
Name [2] 2061 0
Leukaemia Foundation
Address [2] 2061 0
Country [2] 2061 0
Primary sponsor type
Other Collaborative groups
Australasian Leukaemia and Lymphoma Group
Secondary sponsor category [1] 1867 0
Name [1] 1867 0
Address [1] 1867 0
Country [1] 1867 0

Ethics approval
Ethics application status
Ethics committee name [1] 3831 0
Peter MacCallum Cancer Centre
Ethics committee address [1] 3831 0
Ethics committee country [1] 3831 0
Date submitted for ethics approval [1] 3831 0
Approval date [1] 3831 0
Ethics approval number [1] 3831 0

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 tolerability of the combination of these two agents in MDS, with responses at least as good as single agent 5-azacitidine, and to investigate further the mechanisms by which these drugs work and whether we can predict which patients may be more likely to respond.
Trial website
Trial related presentations / publications
Melita Kenealy, Nigel Patton, Robin Filshie, Andrew Nicol, Shir-Jing Ho,
Mark Hertzberg, Tony Mills, Ian Prosser, Emma Link, Linda Cowan, Diana Zannino & John F.
Seymour (2016): Results of a phase II study of thalidomide and azacitidine in patients with
clinically advanced myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia
(CMML) and low blast count acute myeloid leukemia (AML), Leukemia & Lymphoma, DOI:
Public notes

Principal investigator
Name 27868 0
Address 27868 0
Country 27868 0
Phone 27868 0
Fax 27868 0
Email 27868 0
Contact person for public queries
Name 10954 0
Dr Melita Kenealy
Address 10954 0
c/o Department of Haematology and Medical Oncology
Peter MacCallum Cancer Centre
Locked Bag 1
A’Beckett Street
Melbourne VIC 8006
Country 10954 0
Phone 10954 0
+61 3 96561111
Fax 10954 0
+61 3 96561408
Email 10954 0
Contact person for scientific queries
Name 1882 0
Dr Melita Kenealy
Address 1882 0
cc/o Department of Haematology and Medical Oncology
Peter MacCallum Cancer Centre
Locked Bag 1
A’Beckett Street
Melbourne VIC 8006
Country 1882 0
Phone 1882 0
+61 3 96561111
Fax 1882 0
+61 3 96561408
Email 1882 0

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary