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Trial registered on ANZCTR


Registration number
ACTRN12619001532190
Ethics application status
Approved
Date submitted
14/10/2019
Date registered
6/11/2019
Date last updated
6/11/2019
Date data sharing statement initially provided
6/11/2019
Type of registration
Retrospectively registered

Titles & IDs
Public title
The Walter and Eliza Hall (WEHI) Stafford Fox Rare Cancer Program (SFRCP) will use blood and tumour tissue samples from patients with rare cancers to study these types of cancers in detail.
Scientific title
WEHI Stafford Fox Rare Cancer Program.
Discovery of novel therapeutic targets within the biology of rare cancers.
Secondary ID [1] 298479 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rare cancer 313254 0
Condition category
Condition code
Cancer 311695 311695 0 0
Any cancer

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
The purpose of the WEHI Stafford Fox Rare Cancer Program is to use blood and tumour tissue samples from patients with rare cancers to study these types of cancers in detail. Understanding how rare cancers develop and how best to treat them may help doctors to better select appropriate treatment options for future patients.

After enrolment, data will be collected about patients' medical histories including details about the cancer, when/how it was diagnosed, the kinds of cancer treatments given, and progress on these treatments. This may include information on medical file including results of past scans and any molecular or special testing previously performed on the cancer, including genetic tests. Information about other health problems and health information about patient's relatives relating to cancer or genetic conditions that run in the family may be collected.

Patients will be asked to provide up to three blood samples per year for gene sequencing and
tumour tissue samples for gene sequencing and analysis of proteins. The tissue samples may have been previously collected and archived in a hospital laboratory or tissue bank, or may be taken as a fresh tumour sample if surgery is recommended as part of the treatment of the cancer.

Patients will be asked fill out one brief questionnaire about their ability to perform daily activities which can be done at the hospital or by mail.

Patients will then be contacted by their research doctor on a regular basis (every six-twelve months) to ask about their general health, if their disease has worsened, and if they have started any new anti-cancer treatments.

Patients will be enrolled in the WEHI SFRCP over a period of 5 years.
Intervention code [1] 314730 0
Not applicable
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 320413 0
The discovery of novel mechanisms of regulation of rare cancers through basic science research. The Primary Endpoint is discovery. Tumour and blood samples will be analysed by designated laboratories and will be dependent on individual case characteristics. The research design will be adaptive and the outcome is exploratory.
Timepoint [1] 320413 0
At 5 years or after patient accrual is completed whichever occurs first.
Secondary outcome [1] 371510 0
The proportion of rare cancers analysed that are found to have a potential driver event by DNA sequencing or RNASeq expression or immune profiling analysis (current conventional research testing available as a result of common cancer analyses). Tumour and blood samples will be analysed by designated laboratories and will be dependent on individual case characteristics. The research design will be adaptive and the outcome is exploratory.
Timepoint [1] 371510 0
At 5 years or after patient accrual is completed whichever occurs first.
Secondary outcome [2] 371511 0
The proportion of rare cancers analysed that are found to have an observable potential driver event found by more novel analysis methods. Tumour and blood samples will be analysed by designated laboratories and will be dependent on individual case characteristics. The research design will be adaptive and the outcome is exploratory.
Timepoint [2] 371511 0
At 5 years or after patient accrual is completed whichever occurs first.
Secondary outcome [3] 371512 0
The number of novel driver or regulatory events identified using research techniques such as DNA sequencing, RNA expression or immune profiling in rare cancers. Tumour and blood samples will be analysed by designated laboratories and will be dependent on individual case characteristics. The research design will be adaptive and the outcome is exploratory.
Timepoint [3] 371512 0
Secondary outcomes related to individual or related cases will be reported when available.
Secondary outcome [4] 371513 0
The variation in the degree of evolution of molecular heterogeneity across rare cancers. Tumour and blood samples will be analysed by designated laboratories and will be dependent on individual case characteristics. The research design will be adaptive and the outcome is exploratory.
Timepoint [4] 371513 0
At 5 years or after patient accrual is completed whichever occurs first.
Secondary outcome [5] 371514 0
The biology that underlies "super-responders" to current standard and novel therapies in rare cancers. Tumour and blood samples will be analysed by designated laboratories and will be dependent on individual case characteristics. The research design will be adaptive and the outcome is exploratory.
Timepoint [5] 371514 0
At 5 years or after patient accrual is completed whichever occurs first.
Secondary outcome [6] 376104 0
To evaluate psychosocial well-being using a validated brief measure of post-traumatic stress syndrome (the Impact of Event Scale 6 Questionnaire), relevant for the uncertainty of having a rare cancer. Patients may choose to complete a questionnaire at study entry.
Timepoint [6] 376104 0
Assessed at study entry.

Eligibility
Key inclusion criteria
- Aged 18 years or older, with signed informed consent and ability to comply with the protocol.
- Access to tumour tissue must be available from core biopsy or surgical resection from a disease site (cytology alone is insufficient for diagnosis).
- Histologically confirmed rare histopathology diagnosis according to the RARECARE group definition, < 6 per 100,000 population incidence per year (Gatta, 2011).
- Malignancy where little evidenced-based care or standard of care therapies exist.
- Tumour type associated with a poor outcome (patients with a very good prognosis cancer type will not be prioritized for this study).
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Patients who are unable or unwilling, to consent to the study.
- Patients for whom no archival tissue (surgical excision or biopsy) is available.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Both
Statistical methods / analysis
The very nature of rare cancer means that it is impossible to apply statistical methodologies as every patient is unique. A pragmatic approach of enrolling a total sample size of 1250 over 5 years has been taken to provide a reasonable estimate of the variability of rare tumour analysis that we are likely to see in subsequent larger studies. Because this is an exploratory study, a minimum threshold for statistical significance has not been set..

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 13996 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment hospital [2] 13997 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment postcode(s) [1] 26775 0
3000 - Melbourne
Recruitment postcode(s) [2] 26776 0
3050 - Parkville

Funding & Sponsors
Funding source category [1] 303024 0
Charities/Societies/Foundations
Name [1] 303024 0
Stafford Fox Medical Research Foundation
Address [1] 303024 0
1G Royal Parade
Parkville Victoria 3052
Country [1] 303024 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Walter and Eliza Hall Institute of Medical Research
Address
1G Royal Parade
Parkville Victoria 3052
Country
Australia
Secondary sponsor category [1] 304126 0
None
Name [1] 304126 0
Address [1] 304126 0
Country [1] 304126 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 303573 0
Melbourne Health Human Research Ethics Committee
Ethics committee address [1] 303573 0
PO Royal Melbourne Hospital
Parkville VIctoria 3050
Ethics committee country [1] 303573 0
Australia
Date submitted for ethics approval [1] 303573 0
25/11/2015
Approval date [1] 303573 0
31/03/2016
Ethics approval number [1] 303573 0
HREC/15/MH/396
Ethics committee name [2] 304461 0
St John of God Health Care Human Research Ethics Committee
Ethics committee address [2] 304461 0
Suite 304
25 McCourt Street
Subiaco WA 6008
Ethics committee country [2] 304461 0
Australia
Date submitted for ethics approval [2] 304461 0
26/10/2017
Approval date [2] 304461 0
10/11/2017
Ethics approval number [2] 304461 0
1294

Summary
Brief summary
The purpose of the Walter and Eliza Hall Institute Stafford Fox Rare Cancer Program is to use blood and tumour tissue samples from patients with rare cancers to study these types of cancers in detail.

Who is it for?
You may be eligible for this study if you have cancer that is considered rare, occurring in less than 6 people for every 100,000 diagnosed per year. There are literally hundreds of different types of rare or less common cancers. In fact most cancers, except breast, prostate, bowel, lung and skin melanoma, can be considered rare or less common. Examples are soft tissue cancers (sarcomas), brain tumours, cervical cancers, thyroid cancers, liver cancers, testicular cancers and many more. The researchers are also interested if a person has had three or more different types of cancers in their lifetime.

Study details
By consenting to this study, all participants may choose to donate suitable tissue (blood and tumour tissue) for analysis which will help us to better understand the biology driving the development of their rare cancer/s. Participants in this study will then be contacted by their research doctor on a regular basis (every six-twelve months) to ask about their general health for up to 5 years.

We hope that in doing these analyses, novel regulatory mechanisms could be discovered which will allow more effective treatments to be developed.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 94130 0
Prof Clare Scott
Address 94130 0
Walter and Eliza Hall Institute of Medical Research
1G Royal Parade Parkville
Victoria 3052
Country 94130 0
Australia
Phone 94130 0
+61385595000
Fax 94130 0
Email 94130 0
rarecancers@wehi.edu.au
Contact person for public queries
Name 94131 0
Ms Briony Milesi
Address 94131 0
Level 7, The Royal Women’s Hospital
Cnr Grattan Street & Flemington Road
Parkville, Victoria 3052
Country 94131 0
Australia
Phone 94131 0
+61383453733
Fax 94131 0
Email 94131 0
rarecancers@wehi.edu.au
Contact person for scientific queries
Name 94132 0
Prof Clare Scott
Address 94132 0
Walter and Eliza Hall Institute of Medical Research
1G Royal Parade Parkville
Victoria 3052
Country 94132 0
Australia
Phone 94132 0
+61385595000
Fax 94132 0
Email 94132 0
rarecancers@wehi.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Patient's tumour type, molecular analysis, treatment history and outcomes
When will data be available (start and end dates)?
Currently available, no end date determined.
Available to whom?
Only researchers who provide an ethically approved methodologically sound proposal, assessed on a case-by-case basis at the discretion of Primary Sponsor.
Available for what types of analyses?
To achieve the aims in the approved proposal.
How or where can data be obtained?
Access subject to approval by Principal Investigator.
rarecancers@wehi.edu.au
What supporting documents are/will be available?
Study protocol
How or where can supporting documents be obtained?
Type [1] 5292 0
Study protocol
Citation [1] 5292 0
Link [1] 5292 0
Email [1] 5292 0
rarecancers@wehi.edu.au
Other [1] 5292 0
Access subject to approval by Principal Investigator.
Attachment [1] 5292 0
Summary results
No Results