Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05504772


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT05504772
Ethics application status
Date submitted
30/01/2022
Date registered
17/08/2022
Date last updated
15/02/2023

Titles & IDs
Public title
Precision Medicine for Every Child With Cancer
Scientific title
Precision Medicine for Every Child With Cancer
Secondary ID [1] 0 0
ZERO2
Universal Trial Number (UTN)
Trial acronym
ZERO2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Childhood Cancer 0 0
Childhood Solid Tumor 0 0
Childhood Brain Tumor 0 0
Childhood Leukemia 0 0
Refractory Cancer 0 0
Relapsed Cancer 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Whole Genome Sequencing
Other interventions - RNA seq
Other interventions - DNA Methylation
Other interventions - Targeted Panel Sequencing
Other interventions - High Throughput Sequencing (in vitro)
Other interventions - Patient Derived Xenograft (PDX)(in vivo)
Other interventions - Liquid Biopsy

High-risk cancers - One of the following two criteria must be met:
Confirmed or suspected high-risk malignancy defined as expected overall survival < 30% based on current literature for the specific cancer
Cancers for which standard therapy would result in unacceptable and severe morbidity (e.g., infantile fibrosarcoma where definitive surgery would require amputation of limb) Note: This does not include HR neuroblastoma at diagnosis as this group of patients have an overall survival =30% and belongs to Cohort 4A.

Rare tumors - At least one of the following three criteria must be met:
A rare tumor of uncertain prognosis due to rarity of disease
A rare tumor with no established treatment strategy
A cancer where routine histopathological examination has not been able to establish a diagnosis
Confirmed histiocytic disorder AND molecular profiling may facilitate diagnosis and/or treatment
Confirmed proliferative vascular or lymphatic malformation AND has failed conventional treatment, e.g., surgery or embolization, OR no appropriate treatment is available AND the disease is organ, limb or life threatening, or debilitating

Primary central nervous system (CNS) tumours - Patient is suspected or confirmed to have a primary CNS tumor, including low and high-grade tumors

Neuroblastoma - Patient is suspected or confirmed to have neuroblastoma 4A: HR neuroblastoma at diagnosis 4B: Non-HR neuroblastoma

Acute myeloid leukemia, myelodysplastic syndrome and other leukemias not classified as ALL - Patient is confirmed by flow cytometry to have acute myeloid leukemia (AML) or other leukemias (Note: Verbal confirmation of flow cytometry result is adequate for enrolment)

Acute lymphoblastic leukemia (ALL) - Patient is confirmed to have acute lymphoblastic leukemia by flow cytometry (Note: Verbal confirmation of flow cytometry result is adequate for enrolment)

Lymphomas - Patient is suspected or confirmed to have a lymphoma

Sarcomas - Patient is suspected or confirmed to have a sarcoma Includes gastrointestinal stromal tumour (GIST), malignant peripheral nerve sheath tumour (MPNST), desmoplastic small round cell tumour (DSRCT)

Renal tumors - Patient is suspected or confirmed to have a renal tumor Includes clear cell sarcoma of kidney

Hepatic and biliary tree tumors - Patient is suspected or confirmed to have a liver or biliary tree tumor

Thyroid and endocrine tumors - Patient is suspected or confirmed to have a thyroid or endocrine cancer

Other tumors - Patient is suspected or confirmed to have a tumor which does not fit into any of the above

Germline only - One of the following two criteria must be met:
Patients whose submitted tumor sample could not yield sufficient DNA for any molecular analysis AND participants/parents have consented to return of germline findings.
Patients who do not have appropriate tumor sample to be submitted for molecular profiling may be considered for germline only analysis. Obtaining tumor samples wherever possible will be encouraged.


Other interventions: Whole Genome Sequencing
Each tumor sample will be sequenced and analyzed in parallel with its matched normal (germline DNA from the same patient) to enable the identification of somatic aberrations.

Other interventions: RNA seq
Results will be used for bioinformatics analysis for fusion transcripts and gene expression.

Other interventions: DNA Methylation
Genome-wide assessment of DNA methylation will be conducted on all samples where possible.

Other interventions: Targeted Panel Sequencing
Targeted panel sequencing may be performed:
When WGS is not feasible or appropriate, e.g., insufficient DNA from fresh or frozen sample or only Formalin-Fixed Paraffin-Embedded (FFPE) material is available
When mosaicism is suspected
When indicated for a disease type

Other interventions: High Throughput Sequencing (in vitro)
High throughput drug screening will be attempted for tumors from Cohort 1 (high-risk cancers with survival <30%) and selected tumor types.

Other interventions: Patient Derived Xenograft (PDX)(in vivo)
In vivo drug testing in patient derived xenograft (PDX) will be attempted for tumors from Cohort 1 (high-risk cancers) and selected tumor types.

Other interventions: Liquid Biopsy
Liquid biopsy will be investigated as a non-invasive method for diagnosis of tumors that are difficult to biopsy directly, understanding tumor heterogeneity, monitoring of treatment response, and detection of minimal residual disease (MRD)/relapse in leukemia, solid and CNS tumors.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Utility of recommended personalized therapy for HR childhood cancer patients.
Timepoint [1] 0 0
5 years
Primary outcome [2] 0 0
Utility of recommended personalized therapy for non-HR childhood cancer patients.
Timepoint [2] 0 0
5 years
Secondary outcome [1] 0 0
Utility of pre-defined virtual molecular panel for non-HR childhood cancer patients.
Timepoint [1] 0 0
5 years
Secondary outcome [2] 0 0
Utility of comprehensive precision medicine for patients with rare tumors in childhood.
Timepoint [2] 0 0
5 years
Secondary outcome [3] 0 0
Utility of Molecular Tumour Board (MTB) recommendation tier system for HR childhood cancer patients.
Timepoint [3] 0 0
5 years
Secondary outcome [4] 0 0
Utility of preclinical testing in HR childhood cancer patients.
Timepoint [4] 0 0
5 years
Secondary outcome [5] 0 0
Clinical utility of germline WGS in patients with childhood cancers.
Timepoint [5] 0 0
5 years
Secondary outcome [6] 0 0
Treatment outcome in HR childhood cancer patients who have received recommended personalised therapy which are molecularly and/or preclinically directed.
Timepoint [6] 0 0
5 years

Eligibility
Key inclusion criteria
1. Age < 18 years Note: Individual patients aged 19 - 25 years old with a pediatric
cancer, e.g., neuroblastoma, may be enrolled after discussion with, and at the
discretion of, the Study Chair or their delegate.

2. Life expectancy >6 weeks at time of enrolment

3. Consent i. Signed and dated informed consent for study enrolment from participant aged
= 18 years or from parent/guardian of participant aged <18 years. ii. Separate signed
and dated informed consent for understanding the role of germline testing and choice
for the return of germline results.
Minimum age
0 Years
Maximum age
25 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2030
Actual
Sample size
Target
3500
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's and Children's Hospital - Adelaide
Recruitment hospital [2] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [3] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [4] 0 0
Monash Children's Hospital - Melbourne
Recruitment hospital [5] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [6] 0 0
John Hunter Children's Hospital - Newcastle
Recruitment hospital [7] 0 0
Perth Children's Hospital - Perth
Recruitment hospital [8] 0 0
Sydney Children's Hospital, Randwick - Sydney
Recruitment hospital [9] 0 0
The Children's Hospital at Westmead - Sydney
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Hobart
Recruitment postcode(s) [4] 0 0
- Melbourne
Recruitment postcode(s) [5] 0 0
- Newcastle
Recruitment postcode(s) [6] 0 0
- Perth
Recruitment postcode(s) [7] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Other
Name
Australian & New Zealand Children's Haematology/Oncology Group
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Children's Cancer Institute (CCI)
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Minderoo Foundation
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Medical Research Future Fund
Address [3] 0 0
Country [3] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
To improve outcomes for childhood cancer patients through the implementation of precision
medicine.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05504772
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
David Ziegler
Address 0 0
SCHN
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
National Study Coordinator
Address 0 0
Country 0 0
Phone 0 0
+61 2 9382 3102
Fax 0 0
Email 0 0
SCHN-ZERO2@health.nsw.gov.au
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05504772

Additional trial details provided through ANZCTR
Accrual to date
Recruiting in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 103
Sydney Children's Hospital
Recruitment hospital [2] 104
The Children's Hospital at Westmead
Recruitment hospital [3] 105
John Hunter Children's Hospital
Recruitment hospital [4] 106
The Royal Childrens Hospital
Recruitment hospital [5] 107
Monash Children’s Hospital
Recruitment hospital [6] 108
Queensland Children's Hospital
Recruitment hospital [7] 109
Womens and Childrens Hospital
Recruitment hospital [8] 110
Perth Children's Hospital
Recruitment hospital [9] 111
Royal Hobart Hospital
Recruitment postcode(s) [1] 105
2031
Recruitment postcode(s) [2] 106
2145
Recruitment postcode(s) [3] 107
2305
Recruitment postcode(s) [4] 108
3052
Recruitment postcode(s) [5] 109
3168
Recruitment postcode(s) [6] 110
4101
Recruitment postcode(s) [7] 111
5006
Recruitment postcode(s) [8] 112
6009
Recruitment postcode(s) [9] 113
7000
Recruiting in New Zealand
Province(s)/district(s)
Auckland 1023 Christchurch 8011
Funding & Sponsors
Funding source category [1] 80
Government body
Name [1] 80
MRFF Emerging Priorities and Consumer Driven Research
Address [1] 80
Department of Health and Aged Care GPO Box 9848 Canberra ACT 2601
Country [1] 80
Australia
Funding source category [2] 81
Charities/Societies/Foundations
Name [2] 81
Minderoo Foundation’s Collaborate Against Cancer Initiative
Address [2] 81
PO Box 3155 Broadway Nedlands WA 6009
Country [2] 81
Australia
Primary sponsor
Other Collaborative groups
Primary sponsor name
ANZCHOG
Primary sponsor address
Level 6, TRF Building
Hudson Institute
27-31 Wright St
Clayton VIC 3168
Primary sponsor country
Australia
Ethics approval
Ethics application status
Approved
Ethics committee name [1] 55
Sydney Children's Hospitals Network Human Research Ethics Committee
Address [1] 55
Locked Bag 4001 Westmead 2145 NSW
Country [1] 55
Australia
Date submitted for ethics approval [1] 55
24/06/2022
Approval date [1] 55
01/07/2022
Ethics approval number [1] 55
2022/ETH01232
 
Public notes

Contacts
Principal investigator
Title 353 0
Prof
Name 353 0
David Ziegler
Address 353 0
Kids Cancer Centre Sydney Children’s Hospital Randwick, NSW 2031
Country 353 0
Australia
Phone 353 0
+61 (02) 9382 1730
Fax 353 0
+61 (02) 9382 1789
Email 353 0
D.Ziegler@unsw.edu.au
Contact person for public queries
Title 354 0
Name 354 0
KOALA NCC Team
Address 354 0
Kids Cancer Centre Sydney Children’s Hospital Randwick, NSW 2031
Country 354 0
Australia
Phone 354 0
+61 (02) 9382 1730
Fax 354 0
+61 (02) 9382 1789
Email 354 0
SCHN-ZERO2@health.nsw.gov.au
Contact person for scientific queries
Title 355 0
Prof
Name 355 0
David Ziegler
Address 355 0
Kids Cancer Centre Sydney Children’s Hospital Randwick, NSW 2031
Country 355 0
Australia
Phone 355 0
+61 (02) 9382 1730
Fax 355 0
+61 (02) 9382 1789
Email 355 0
D.Ziegler@unsw.edu.au