ANZCTR - Registration

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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers

Trial registered on ANZCTR

Registration number

ACTRN12607000283471

Ethics application status

Approved

Date submitted

17/05/2007

Date registered

28/05/2007

Date last updated

4/08/2023

Date data sharing statement initially provided

18/06/2020

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]

Australasian Leukaemia and Lymphoma Group (ALLG): ALLG MDS3

Universal Trial Number (UTN)

Trial acronym

nil

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Myelodysplastic Syndromes

Condition category

Condition code

Blood

Other blood disorders

Intervention/exposure

Study type

Interventional

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]

Treatment: Drugs

Comparator / control treatment

Control group

Uncontrolled

Outcomes

Primary outcome [1]

Haematologic and Non-haematologic toxicity of the combination of thalidomide and 5-azacitidine.

Timepoint [1]

Toxicities are measured as they occur throughout the study period.

Secondary outcome [1]

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]

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]

Leukaemic transformation or death

Timepoint [2]

Is recorded as it occurs throughout the study period.

Secondary outcome [3]

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]

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.

Eligibility

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

Sex

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

Treatment

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

Phase 1 / Phase 2

Type of endpoint/s

Safety/efficacy

Statistical methods / analysis

Recruitment

Recruitment status

Completed

Date of first participant enrolment

Anticipated

30/06/2007

Actual

1/07/2008

Date of last participant enrolment

Anticipated

Actual

3/07/2009

Date of last data collection

Anticipated

Actual

5/09/2012

Sample size

Target

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

Funding & Sponsors

Funding source category [1]

Commercial sector/Industry

Name [1]

Pharmion Australia

Country [1]

Australia

Funding source category [2]

Charities/Societies/Foundations

Name [2]

Leukaemia Foundation

Country [2]

Australia

Primary sponsor type

Other Collaborative groups

Name

Australasian Leukaemia and Lymphoma Group

Country

Australia

Secondary sponsor category [1]

None

Name [1]

nil

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Peter MacCallum Cancer Centre

Ethics committee address [1]

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

Approval date [1]

14/05/2007

Ethics approval number [1]

07/07

Summary

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:
10.1080/10428194.2016.1190971

Public notes

Contacts

Principal investigator

Name

Address

Country

Phone

Fax

Contact person for public queries

Name

Dr Melita Kenealy

Address

c/o Department of Haematology and Medical Oncology Peter MacCallum Cancer Centre Locked Bag 1 A’Beckett Street Melbourne VIC 8006

Country

Australia

Phone

+61 3 96561111

Fax

+61 3 96561408

Melita.Kenealy@petermac.org

Contact person for scientific queries

Name

Dr Melita Kenealy

Address

cc/o Department of Haematology and Medical Oncology Peter MacCallum Cancer Centre Locked Bag 1 A’Beckett Street Melbourne VIC 8006

Country

Australia

Phone

+61 3 96561111

Fax

+61 3 96561408

Melita.Kenealy@petermac.org

Data sharing statement

Will the study consider sharing individual participant data?

Yes

Will there be any conditions when requesting access to individual participant data?

Persons/groups eligible to request access:
• 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.

Conditions for requesting access:
• -

What individual participant data might be shared?

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

What types of analyses could be done with individual participant data?

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

When can requests for individual participant data be made (start and end dates)?

From:
Data available 3 months following publication, for an indefinite period

To:
-

Where can requests to access individual participant data be made, or data be obtained directly?

• Access can be requested via the Health Data Australia catalogue (https://researchdata.edu.au/health/). Search for the ACTRN number in the catalogue to find datasets associated with this trial or email enquiries to info@allg.org.au

Are there extra considerations when requesting access to individual participant data?

What supporting documents are/will be available?

Doc. No.	Type	Citation	Link	Email	Other Details	Attachment
19919	Study protocol			info@allg.org.au	Access can be requested via the Health Data Austra... [More Details]

Results publications and other study-related documents

Documents added manually

No documents have been uploaded by study researchers.

Documents added automatically

Source	Title	Year of Publication	DOI
Embase	Hemoglobin is a key determinant of quality of life before and during azacitidine-based therapy for myelodysplasia and low blast count acute myeloid leukemia.	2022	https://dx.doi.org/10.1080/10428194.2021.2012664

N.B. These documents automatically identified may not have been verified by the study sponsor.

Trial Review

Documents added manually

Documents added automatically