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Trial registered on ANZCTR


Registration number
ACTRN12616001000493
Ethics application status
Approved
Date submitted
22/07/2016
Date registered
28/07/2016
Date last updated
24/10/2019
Date data sharing statement initially provided
16/11/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
A study to evaluate the feasibility of performing genomic testing of rare cancers to match the cancer to treatment. GeNOmic MatchINg treATment fOr Rare cancers (NOMINATOR).
Scientific title
A national multicentre clinical trial to assess the feasibility of performing genomic testing of rare cancers to match the cancer to treatment.
Secondary ID [1] 289741 0
Nil known
Universal Trial Number (UTN)
Trial acronym
GeNOmic MatchINg treATment fOr Rare cancers (NOMINATOR).
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rare cancer. 299585 0
Condition category
Condition code
Cancer 299555 299555 0 0
Any cancer

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Eligible participants will consent to the collection of tumour tissue and up to three blood samples. If clinically indicated, a tumour biopsy will be performed for some participants, or an archival sample of the tumour will be retrieved for a gene sequencing test. The study will use gene sequencing to identify genetic mutations present in a person’s cancer cells (a cancer genomic profile). Following analysis of the samples, each case will be discussed in a Molecular Tumour Board with the involvement of a range of relevant experts, including scientists, bioinformaticians, specialists in genetics and oncologists. The Molecular Tumour Board will comment on validated or promising treatments as a result of the genomic analysis - these will not be limited to locally available or PBS funded medications. Recommendations are non-prescriptive and the patient’s treating oncologist will decide on treatment options. Results of the gene sequencing test will be sent to the participant’s treating oncologist.

Participants may elect to receive the results of the gene sequencing test from their treating oncologist, or to attend a visit at the hospital. After the results have been discussed, participants will be asked to fill out questionnaires that will ask about participation in the study, and whether or not they feel that the gene sequencing test has been helpful in terms of choosing future treatment options.

Participants will be asked to:
*attend a screening visit to have some tests done to assess whether or not the NOMINATOR study is suitable for them;
*attend one or more hospital visits to collect blood and tumour samples and complete a baseline questionnaire;
*attend an optional hospital visit to receive the results of the gene sequencing test and to complete a questionnaire;
*attend up to two more visits to the hospital for blood tests which will be used to track circulating tumour DNA which may help identify response to treatment (this analysis is exploratory).
*complete questionnaires at 6, 12 and at 24 months (participants may complete questionnaires during hospital visits or by mail).
Intervention code [1] 295379 0
Diagnosis / Prognosis
Comparator / control treatment
No control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 299033 0
To determine the proportion of rare cancer cases where a potentially actionable aberration (pathogenic mutation vs no mutation vs possible mutation, ie level of evidence reported) is identified following analysis of genetic sequencing.
Timepoint [1] 299033 0
Level of evidence reported will be assessed by the Molecular Tumour Board following genetic sequencing of each participant’s tumour biopsy sample..
Secondary outcome [1] 325926 0
The proportion of rare cancer cases for which a relevant drug or clinical trial is available (regardless of access).
Timepoint [1] 325926 0
Determined by Molecular Tumour Board review/recommendation and search of clinical trial registries. Medical record data will also be collected and assessed after the last participant is enrolled.
Secondary outcome [2] 325927 0
The proportion of cases for which a relevant drug or trial is available in the relevant Australian state.
Timepoint [2] 325927 0
Determined by Molecular Tumour Board review/recommendation and search of clinical trial registries. Data from investigators, recruitment and medical records will also be collected and assessed after the last participant is enrolled.
Secondary outcome [3] 325928 0
The number of cases in which a relevant trial or drug is accessed.
Timepoint [3] 325928 0
Data will be collected from investigators and medical records and assessed after the last participant is enrolled.
Secondary outcome [4] 325929 0
The proportion of cases where genetic sequencing testing impacted on patient management.
Timepoint [4] 325929 0
Data will be collected from investigators and medical records and assessed after the last participant is enrolled.
Secondary outcome [5] 325930 0
The proportion of cases where molecular testing impacted on understanding of etiology of the patient’s rare cancer, including a change in tumour diagnosis.
Timepoint [5] 325930 0
Data will be collected from investigators and medical records and assessed after the last participant is enrolled.
Secondary outcome [6] 325931 0
The number of suitable patients across multiple sites.
Timepoint [6] 325931 0
Recruitment data will be collected and assessed after the last participant is enrolled.
Secondary outcome [7] 325932 0
Assess psychosocial well-being, relevant for the uncertainty of having a rare cancer using the validated Impact of Event Scale 6 Questionnaire.
Timepoint [7] 325932 0
Data will collected at baseline, after the results of genomic sequencing report have been received, and at the 6 month, 12 month and 24 month time-points for each participant. Data will be analysed after the last participant is registered.
Secondary outcome [8] 326040 0
Identify the response to treatment, if documented.

Timepoint [8] 326040 0
Medical record data will be collected and assessed after the last participant is enrolled.
Secondary outcome [9] 326041 0
Identify the proportion of patients approached who consent to the study.
Timepoint [9] 326041 0
Recruitment data will be collected and assessed after the last participant is enrolled.

Eligibility
Key inclusion criteria
* Patients aged over 18 years, with signed informed consent and ability to comply with protocol requirements
* Patients with Eastern Cooperative Oncology Group (ECOG) performance status 0-1
* Patients with a life expectance of >12 weeks
* Access to tumour tissue is available from core biopsy or surgical resection from a disease site
* Histologically confirmed rare histopathology diagnosis according to the RARECARE group definition
* Malignancy where little evidence-based care or standard of care therapies exist
* Tumour type associated with a poor outcome
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 who have a concurrent active malignancy other than adequately treated non-melanomatous skin cancer, early prostatic adenocarcinoma treated with curative intent or non-invasive carcinoma / in-situ neoplasm of the cervix or breast. Patients with a previous history of malignancy will be eligible provided they have been disease-free for >5 years.

Study design
Purpose of the study
Diagnosis
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
Not Applicable
Type of endpoint(s)
Efficacy
Statistical methods / analysis
This is an exploratory study which aims to provide a reasonable estimate of the variability of rare tumour analysis that we are likely to see in subsequent larger studies. A minimum for statistical significance has not been set and the analysis will be descriptive.

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)
QLD,SA,WA,VIC
Recruitment hospital [1] 6238 0
The Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 6239 0
Royal Brisbane & Womens Hospital - Herston
Recruitment hospital [3] 6240 0
St John of God Hospital, Subiaco - Subiaco
Recruitment hospital [4] 8669 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment postcode(s) [1] 13659 0
5000 - Adelaide
Recruitment postcode(s) [2] 13660 0
4029 - Herston
Recruitment postcode(s) [3] 13661 0
6008 - Subiaco
Recruitment postcode(s) [4] 16781 0
3000 - Melbourne

Funding & Sponsors
Funding source category [1] 294122 0
Charities/Societies/Foundations
Name [1] 294122 0
Stafford Fox Medical Research Foundation
Address [1] 294122 0
Walter and Eliza Hall Institute
1G Royal Parade, Parkville VIC 3052
Country [1] 294122 0
Australia
Funding source category [2] 294123 0
Other Collaborative groups
Name [2] 294123 0
Melbourne Genomics Health Alliance
Address [2] 294123 0
Walter and Eliza Hall Institute
1G Royal Parade, Parkville VIC 3052
Country [2] 294123 0
Australia
Primary sponsor type
Hospital
Name
Melbourne Health
Address
Melbourne Health Office for Research
PO Royal Melbourne Hospital
Parkville Victoria 3050
Country
Australia
Secondary sponsor category [1] 292950 0
None
Name [1] 292950 0
Address [1] 292950 0
Country [1] 292950 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 295528 0
Melbourne Health Human Research Ethics Committee
Ethics committee address [1] 295528 0
PO Royal Melbourne Hospital
Parkville Victoria 3050
Ethics committee country [1] 295528 0
Australia
Date submitted for ethics approval [1] 295528 0
29/09/2015
Approval date [1] 295528 0
14/12/2015
Ethics approval number [1] 295528 0
HREC/15/MH/310

Summary
Brief summary
The primary purpose of the NOMINATOR research study is to determine if new techniques for gene sequencing can be used in the treatment of rare cancers.

Who is it for?
You may be eligible to participate in this trial if you are aged 18 or over and have been diagnosed with a rare cancer which has few standard treatment options and is associated with poor outcome.

Study details:
All participants enrolled in this trial will provide samples of blood and tumour tissue either via retrieval of stored tumour sample from another lab/biobank if this is available or by undergoing a biopsy procedure to obtain fresh samples of tumour. These samples will be used for the gene sequencing test. We will also collect health information from the participant (either via direct questioning by their doctor or from their medical records), in order to help researchers interpret the genomic sequencing results which are obtained from testing. A panel of cancer experts will review the genomic sequencing results and provide advice about treatments that could potentially be more effective as a result of those findings. The patient will then have the option to receive the results of this testing (this is optional), and their doctor may be able to use these tests results to better determine an effective treatment and management plan for their disease.

It is hoped that understanding a cancer’s genomic profile may help doctors to select appropriate treatment options, according to the particular genomic profile of that person’s cancer. This trial will provide information on whether this is feasible in practice.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 67638 0
A/Prof Clare Scott
Address 67638 0
Walter and Eliza Hall Institute of Medical Research
Walter and Eliza Hall Institute
1G Royal Parade
Parkville Victoria 3052
Australia


Country 67638 0
Australia
Phone 67638 0
+61 3 9345 2498
Fax 67638 0
+61 3 9347 0852
Email 67638 0
nominator@mh.org.au
Contact person for public queries
Name 67639 0
Dr Damien Kee
Address 67639 0
Walter and Eliza Hall Institute
1G Royal Parade
Parkville Victoria 3052
Australia
Country 67639 0
Australia
Phone 67639 0
+61 3 93452981
Fax 67639 0
Email 67639 0
nominator@mh.org.au
Contact person for scientific queries
Name 67640 0
Dr Damien Kee
Address 67640 0
Walter and Eliza Hall Institute
1G Royal Parade
Parkville Victoria 3052
Australia
Country 67640 0
Australia
Phone 67640 0
+61 3 93452981
Fax 67640 0
Email 67640 0
nominator@mh.org.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Further discussion required with trial principal investigator.
What supporting documents are/will be available?
No other documents available
Summary results
No Results