Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622000410752
Ethics application status
Approved
Date submitted
1/12/2021
Date registered
10/03/2022
Date last updated
4/08/2023
Date data sharing statement initially provided
10/03/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Advancing treatment therapies in Myelodysplasia
Scientific title
MDS05: Advancing therapies in Myelodysplasia: . A multi-domain platform trial for patients with myelodysplasia investigating new treatments - The Master Protocol
Secondary ID [1] 305941 0
MDS05
Universal Trial Number (UTN)
Trial acronym
MYDAS-T
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplasia 324538 0
Condition category
Condition code
Cancer 322008 322008 0 0
Leukaemia - Acute leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The MYDAS-T study is a multi-domain platform trial. This master protocol will govern the development and management of an adaptive clinical trial platform that will incorporate novel therapeutic combinations for medium and high risk patients with MDS.

The main objective is to assess safety and tolerability of these new treatment combinations for this in need patient population.

The overall durations of the treatment domains will range from 5-10 years depending on the treatment combination being trialed the required follow up period length.

The therapies trialed in this domain will be new and established investigational medicinal products that may range from chemotherapy to biologic target therapies.

Patients will be monitored at their standard of care clinic visits and associated tests eg bone marrow aspiration, blood tests, physical examinations.

Depending on the number of domains open, patients may be stratified or randomised to a domain based on strata such as MDS risk and age. The allocation will be dependent on the number of domains available at the time of patient entry.
Intervention code [1] 322335 0
Treatment: Drugs
Comparator / control treatment
NA
Control group
Uncontrolled

Outcomes
Primary outcome [1] 329763 0
Event free survival will be assessed by analysing morphological blood and bone marrow markers of disease response eg white cell count and blast count.
Timepoint [1] 329763 0
. The endpoints are likely to be assessed for each treatment domain when all patients have completed the domain specific treatment period and trial follow up period. This could be 2-5 years depending on the trial domain.
Primary outcome [2] 329764 0
Overall response rate will be assessed by analysing morphological blood and bone marrow markers of disease response eg white cell counts and blast count.
Timepoint [2] 329764 0
The endpoints are likely to be assessed for each treatment domain when all patients have completed the domain specific treatment period and trial follow up period. This could be 2-5 years depending on the trial domain.
Secondary outcome [1] 403685 0
Overall survival will be assessed by reviewing patient medical records.
Timepoint [1] 403685 0
The endpoints are likely to be assessed for each treatment domain when all patients have completed the domain specific treatment period and trial follow up period. This could be 2-5 years depending on the trial domain.
Secondary outcome [2] 403686 0
Transfusion independence will be assessed using blood count markers, eg whether a patient blood count infers a transfusion is required.
Timepoint [2] 403686 0
The endpoints are likely to be assessed for each treatment domain when all patients have completed the domain specific treatment period and trial follow up period. This could be 2-5 years depending on the trial domain.
Secondary outcome [3] 403687 0
Quality of life using the EORTC QLQ 30 questionnaire will be completed by the enrolled patients.
Timepoint [3] 403687 0
Questionnaires will be completed at baseline, 6, 12, 18, and 24 months of treatment with results analysed at the end of the treatment period.

Eligibility
Key inclusion criteria
1. Provision of written informed consent
2. Provision of written informed consent to the ALLG NBCR
3. Age 18+ (Age 16-17 permitted if consent for minor PICF approved by the authorising HREC)
4. A diagnosis of MDS or AML with less than 30% blasts
Minimum age
16 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History of other malignancy requiring active systemic treatment, or which is likely to result in an expected survival time of less than 12 months.
2. Viral infection with known HIV or viral hepatitis type B or C not adequately controlled by antiviral medication
3. Prior bone marrow or stem cell transplantation for a diagnosis of Myelodysplasia or acute myeloid leukaemia. If stem cell transplantation has been undertaken for other reasons- refer to individual domain and discuss with study team.

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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 1 / Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/12/2022
Actual
30/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
1000
Accrual to date
32
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,SA,TAS,WA,VIC
Recruitment outside Australia
Country [1] 24361 0
New Zealand
State/province [1] 24361 0

Funding & Sponsors
Funding source category [1] 310283 0
Other Collaborative groups
Name [1] 310283 0
Australasian Leukaemia and Lymphoma Group (ALLG)
Country [1] 310283 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Australasian Leukaemia and Lymphoma Group (ALLG)
Address
Ground Floor, 35 Elizabeth Street,
Richmond
VIC
3121
Country
Australia
Secondary sponsor category [1] 311391 0
None
Name [1] 311391 0
none
Address [1] 311391 0
none
Country [1] 311391 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 309951 0
Central Adelaide Local Health Network Human Research Ethics Committee
Ethics committee address [1] 309951 0
Royal Adelaide Hospital
Port Road,
Adelaide
South Australia 5000
Ethics committee country [1] 309951 0
Australia
Date submitted for ethics approval [1] 309951 0
31/03/2022
Approval date [1] 309951 0
18/08/2022
Ethics approval number [1] 309951 0

Summary
Brief summary
Traditionally, combination therapy has not proven to be effective in Myelodysplasia (MDS) given the increased toxicity of drugs used in combination with standard therapies. The aim of this master platform trial is to test a range of novel treatments aimed at targeting MDS to find safe and effective drug combinations.

You may be eligible to participate in this study if you are aged 16 or older (depending on the study, this may be limited to 18 and older) and you have a diagnosis of either MDS or acute myeloid leukemia (AML) with less than 30% blasts.

If you choose to enrol in this trial you will undergo a screening process where blood and bone marrow samples are taken prior to starting any treatments. The study doctors will assess your samples for specific MDS markers and will then prescribe a combination treatment that may be effective. Participants who do not respond to treatment or whose disease worsens may be removed from treatment and be reassessed for the next best treatment option to receive. During treatment participants will be assessed regularly which will include physical exams, blood tests, ECG (heart monitoring), bone marrow biopsies, toxicities to the treatment, quality of life. After treatment, disease will continue to be monitored and if participants have MDS progression or morphological relapse they will be assessed for the next best treatment option.

It is hoped that the results of this trial will help us to determine which drug combinations are safe (minimal side effects/toxicity) and effective for treating MDS.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 115962 0
A/Prof Anoop K Enjeti
Address 115962 0
Calvary Mater Newcastle
Edith and Platt Streets,
Waratah
NSW
2298
Country 115962 0
Australia
Phone 115962 0
+612 4014 3021
Fax 115962 0
Email 115962 0
[email protected]
Contact person for public queries
Name 115963 0
Ms Delaine Smith
Address 115963 0
ALLG
Ground Floor, 35 Elizabeth Street,
Richmond
VIC
3121
Country 115963 0
Australia
Phone 115963 0
+613 8373 9701
Fax 115963 0
Email 115963 0
[email protected]
Contact person for scientific queries
Name 115964 0
Ms Delaine Smith
Address 115964 0
ALLG
Ground Floor, 35 Elizabeth Street,
Richmond
VIC
3121
Country 115964 0
Australia
Phone 115964 0
+613 8373 9701
Fax 115964 0
Email 115964 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
No individual patient data will be available. All results will be presented as aggregate data.


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.