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Trial registered on ANZCTR


Registration number
ACTRN12625000160437
Ethics application status
Approved
Date submitted
19/11/2024
Date registered
11/02/2025
Date last updated
11/02/2025
Date data sharing statement initially provided
11/02/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
The Molecular Screening and Therapeutics in Leukaemia and Lymphoma Study in participants with haematological malignancies
Scientific title
Evaluate the feasibility and benefits of the Molecular Screening and Therapeutics in Leukaemia and Lymphoma Study, in participants with haematological malignancies
Secondary ID [1] 313423 0
Nil known
Universal Trial Number (UTN)
Trial acronym
MoST-LLy
Linked study record
The study is based on the previous Haematology screening program MoST-LLy within the MoST framework protocol (ACTRN12616000908437), focusing on the previously established haematology screening program

Health condition
Health condition(s) or problem(s) studied:
haematological malignancies 335793 0
Leukaemia 336322 0
Lymphoma 336478 0
Myeloma 336479 0
Myeloproliferative neoplasm (MPN) 336480 0
Myelodysplastic syndrome (MDS) 336481 0
Condition category
Condition code
Cancer 332367 332367 0 0
Leukaemia - Acute leukaemia
Cancer 332368 332368 0 0
Leukaemia - Chronic leukaemia
Cancer 332369 332369 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 332370 332370 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Cancer 332371 332371 0 0
Myeloma
Blood 332372 332372 0 0
Haematological diseases

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
This study will evaluate the feasibility and benefits of a molecular screening platform in participants with haematological malignancies (18 years or over). A national molecular tumour board will send a report to the referring haematologist with information on (i) Any genetic biomarkers that were identified in the blood cancer and (ii) The types of treatment that may be suitable (if any are found).

Eligible patients are invited to consent through the completion of a participant information and consent form (PICF). Participants consent to:
- a sample of their blood cancer sent for molecular screening.
- collection of medical and treatment history
- a short questionnaire (6 questions) to be completed after consent and at 6, 12 & 24months
- a research blood sample collected (can be done locally)
Consent, questionnaires and collection of blood sample may take up to 2 hours.

The national Molecular Tumour Board (MTB) of Haematologists, Genetics specialists and invited referring doctors review the results of the molecular screening via video conferencing on a regular basis (weekly/fortnightly as needed). Reports to referring haematologist are aimed to be sent between 6-8 weeks post consent.
Intervention code [1] 329986 0
Early Detection / Screening
Comparator / control treatment
No control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 339923 0
Primary Outcome 1: The percentage of participants with actionable molecular biomarkers identified in screened patients.
Timepoint [1] 339923 0
This will be monitored throughout the study and assessed at the end of the enrolment period.
Primary outcome [2] 339924 0
Primary Outcome 2: The percentage of participants receiving treatment recommendations on the basis of molecular screening.
Timepoint [2] 339924 0
This will be monitored throughout the study and assessed at the end of the enrolment period.
Primary outcome [3] 339925 0
Primary Outcome 3: Percentage of participants receiving treatment based on recommendation.
Timepoint [3] 339925 0
This will be monitored throughout the study at 6, 12 and 24 months post enrolment, with the final assessment at the end of the study.
Secondary outcome [1] 441941 0
Secondary Outcome 1: The number of participants enrolled into screening over defined periods
Timepoint [1] 441941 0
This will be monitored throughout the study and assessed at the end of the enrolment period
Secondary outcome [2] 441942 0
Secondary Outcome 2: Time taken for molecular screening results to be made available, defined as: the interval from the date of consent to date of MTB report returned to clinician.
Timepoint [2] 441942 0
This will be monitored throughout the study and assessed at the end of the enrolment period.
Secondary outcome [3] 441943 0
Review of assay performance including sequencing failure rate, costs per genotype screened will be assessed as a composite outcome.
Timepoint [3] 441943 0
This will be monitored throughout the study and assessed at the end of the enrolment period after all participants have Haem MTB reporting completed.
Secondary outcome [4] 441944 0
Descriptive list and frequency of all anti-cancer therapies received will be assessed as a composite outcome.
Timepoint [4] 441944 0
This will be monitored throughout the study at 6, 12 and 24 months post enrolment, with the final assessment at the end of the study.
Secondary outcome [5] 441945 0
Secondary Outcome 5: Real-world best overall response (rwBOR): defined as as the percentage of participants with a complete response or partial response assessed by the investigator based on evaluable response assessments performed
Timepoint [5] 441945 0
Medical records and follow up information received by referring clinician at 6, 12 and 24 months.
Secondary outcome [6] 441946 0
Secondary Outcome 6: Real-world progression-free survival (rwPFS) - time during which the patient lives without disease worsening or death
Timepoint [6] 441946 0
This will be monitored throughout the study at 6, 12 and 24 months post enrolment, with the final assessment at the end of the study.
Secondary outcome [7] 441947 0
Secondary Outcome 7: Overall Survival (OS) is defined as the interval from the date of consent to date of death from any cause. Participants who did not die will be censored at the date of last known follow-up alive
Timepoint [7] 441947 0
This will be monitored throughout the study at 6, 12 and 24 months post enrolment, with the final assessment at the end of the study.

Eligibility
Key inclusion criteria
1. Participants, aged 18 years and older, with pathologically confirmed blood cancer at initial diagnosis or relapse/refractory disease.
2. Sufficient and accessible tissue for molecular screening.
3. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.
4. Willing and potentially able to comply with study requirements.
5. Signed, written informed consent to participation in the molecular screening.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Comorbidities or conditions which may contraindicate participation, compromise assessment of key outcomes or limit ability to comply with protocol.
2. Patient is eligible for Medicare Benefits scheme (MBS) funded Next Generation Sequencing (NGS), except where there is a clinical rationale to provide a larger sequencing panel or repeat sequencing.

Study design
Purpose
Screening
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
This is a descriptive study of 300 participants with haematological malignancies (Australia only), to screen and identify actionable genomic alterations with potential treatment options. With 300 participants the width of a 95% confidence interval for a proportion is approximately 0.11 if the proportion is around 0.6. This is a descriptive pilot study without any group comparisons and hypothesis tests.
The descriptive analysis will describe the feasibility, efficiency, and utility of implementation of molecular screening.
Tables will report the number of participants satisfying the various conditions of interest. All the key outcome percentages will be reported with 95% confidence intervals (computed using the Wilson Score method). Durations will be reported as median, range, 25th / 75th percentiles and mean (with standard deviation if approximately normal). Time to event outcomes will be summarised using Kaplan-Meier plots. Results will also be reported stratified by Lymphoma vs Leukaemia, genetic subgroup as well as by treatment pathway received.
The prognostic and potentially predictive value of genomic biomarkers will be examined in the entire cohort, as well as in lymphoma and leukaemia subgroups. Survival and efficacy endpoints associated with therapy linked to genomic profiling will be estimated using both response endpoints and time-to-event analyses.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
7/03/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors
Funding source category [1] 317853 0
Government body
Name [1] 317853 0
Department of Health and AgedCare: Medical Research Future Fund (MRFF)
Country [1] 317853 0
Australia
Funding source category [2] 317854 0
Charities/Societies/Foundations
Name [2] 317854 0
Luekaemia Foundation of Australia
Country [2] 317854 0
Australia
Primary sponsor type
Other
Name
QIMRBerghofer Medical Research Institute
Address
Country
Australia
Secondary sponsor category [1] 320186 0
None
Name [1] 320186 0
Address [1] 320186 0
Country [1] 320186 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316535 0
Metro South Human Research Ethics Committee
Ethics committee address [1] 316535 0
Ethics committee country [1] 316535 0
Australia
Date submitted for ethics approval [1] 316535 0
14/11/2024
Approval date [1] 316535 0
21/01/2025
Ethics approval number [1] 316535 0
HREC/2024/QMS/113451

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 138194 0
Prof Steven Lane
Address 138194 0
QIMR Berghofer Medical Research Institute Locked Bag 2000 Royal Brisbane Hospital, QLD 4029
Country 138194 0
Australia
Phone 138194 0
+61 7 3845 3766
Fax 138194 0
Email 138194 0
Steven.Lane@qimrberghofer.edu.au
Contact person for public queries
Name 138195 0
MoST-LLy project manager
Address 138195 0
QIMR Berghofer Medical Research Institute Locked Bag 2000 Royal Brisbane Hospital, QLD 4029
Country 138195 0
Australia
Phone 138195 0
+61738453678
Fax 138195 0
Email 138195 0
mostlly@qimrberghofer.edu.au
Contact person for scientific queries
Name 138196 0
Prof Steven Lane
Address 138196 0
QIMR Berghofer Medical Research Institute Locked Bag 2000 Royal Brisbane Hospital, QLD 4029
Country 138196 0
Australia
Phone 138196 0
+61733620222
Fax 138196 0
Email 138196 0
mostlly@qimrberghofer.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Currently no plan and participant consent will be required


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.