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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
17/07/2024
Titles & IDs
Public title
Precision Medicine for Every Child With Cancer
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Scientific title
Precision Medicine for Every Child With Cancer
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Secondary ID [1]
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ZERO2
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Universal Trial Number (UTN)
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Trial acronym
ZERO2
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Childhood Cancer
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Childhood Solid Tumor
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Childhood Brain Tumor
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Childhood Leukemia
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Refractory Cancer
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Relapsed Cancer
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0
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Condition category
Condition code
Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Treatment: Other - Whole Genome Sequencing
Treatment: Other - RNA seq
Treatment: Other - DNA Methylation
Treatment: Other - Targeted Panel Sequencing
Treatment: Other - High Throughput Sequencing (in vitro)
Treatment: Other - Patient Derived Xenograft (PDX)(in vivo)
Other interventions - Liquid Biopsy
High-risk cancers - One of the following two criteria must be met:
1. Confirmed or suspected high-risk malignancy defined as expected overall survival \< 30% based on current literature for the specific cancer
2. 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:
1. A rare tumor of uncertain prognosis due to rarity of disease
2. A rare tumor with no established treatment strategy
3. A cancer where routine histopathological examination has not been able to establish a diagnosis
4. Confirmed histiocytic disorder AND molecular profiling may facilitate diagnosis and/or treatment
5. 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:
1. Patients whose submitted tumor sample could not yield sufficient DNA for any molecular analysis AND participants/parents have consented to return of germline findings.
2. 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.
Treatment: Other: 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.
Treatment: Other: RNA seq
Results will be used for bioinformatics analysis for fusion transcripts and gene expression.
Treatment: Other: DNA Methylation
Genome-wide assessment of DNA methylation will be conducted on all samples where possible.
Treatment: Other: Targeted Panel Sequencing
Targeted panel sequencing may be performed:
1. 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
2. When mosaicism is suspected
3. When indicated for a disease type
Treatment: Other: 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.
Treatment: Other: 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.
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Intervention code [1]
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Treatment: Other
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Utility of recommended personalized therapy for HR childhood cancer patients.
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Assessment method [1]
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Disease control rate (stable disease + partial response + complete response) in HR patients who have received recommended personalized therapy which are molecularly and/or preclinically directed
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Timepoint [1]
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5 years
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Primary outcome [2]
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Utility of recommended personalized therapy for non-HR childhood cancer patients.
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Assessment method [2]
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The proportion of non-HR patients for whom the disease specific, clinically relevant, virtual molecular panel provides additional or equivalent results for diagnosis and risk stratification when compared with routine diagnostic tests.
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Timepoint [2]
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5 years
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Secondary outcome [1]
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Utility of pre-defined virtual molecular panel for non-HR childhood cancer patients.
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Assessment method [1]
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The proportion of non-HR patients for whom a pre-defined virtual molecular panel; leads to a streamline molecular report issued within 4 weeks from receipt of samples, changes or refines the initial histopathological diagnosis, changes or refines risk stratification at diagnosis, changes or refines treatment at diagnosis and/or facilitates enrolment in clinical trials requiring prior molecular studies.
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Timepoint [1]
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5 years
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Secondary outcome [2]
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Utility of comprehensive precision medicine for patients with rare tumors in childhood.
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Assessment method [2]
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Proportion of rare cancer cohort patients for which comprehensive precision medicine improves diagnosis, identifies at least one therapeutic target or facilitates improvement in therapy within a clinically relevant timeframe.
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Timepoint [2]
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5 years
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Secondary outcome [3]
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Utility of Molecular Tumour Board (MTB) recommendation tier system for HR childhood cancer patients.
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Assessment method [3]
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Evaluation of the correlation of MTB recommendation tier to treatment outcome, clinician rated value of MTB recommendation tier in facilitating therapeutic decision and drug access and proportion of HR patients for which the MTB recommendation improves diagnosis, risk stratification or facilitates improvement in therapy within a clinically relevant timeframe.
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Timepoint [3]
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5 years
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Secondary outcome [4]
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Utility of preclinical testing in HR childhood cancer patients.
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Assessment method [4]
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Evaluation of proportion of tumors where in vitro sensitivity testing can be successfully performed compared with PRISM trial, turnaround time for preclinical in vitro and in vivo drug testing, proportion of tumors where in vitro drug sensitivity identifies additional molecular drivers and proportion of patients for whom preclinical testing:
i. Facilitates therapeutic decision ii. Identifies additional therapeutic options in patients for whom genomic profiling did not identify molecular targets iii. Predicts clinical outcome
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Timepoint [4]
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5 years
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Secondary outcome [5]
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Clinical utility of germline WGS in patients with childhood cancers.
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Assessment method [5]
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Evaluation of;
1. Proportion of HR and non-HR patients with a reportable germline finding (i. For whom the result was not previously known ii. For whom the results would have been missed using current clinical testing criteria)
2. Proportion of patients for whom medical management for the current cancer and future cancer risk has been altered based on the germline findings
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Timepoint [5]
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5 years
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Secondary outcome [6]
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Treatment outcome in HR childhood cancer patients who have received recommended personalised therapy which are molecularly and/or preclinically directed.
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Assessment method [6]
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Evaluation of;
1. Objective response in patients who have received single agent versus combination personalized therapy
2. Disease control (stable disease + partial response + complete response) in patients who have received a single agent versus combination personalized therapy
3. Progression-free interval (PFI) ratio: PFI personalized therapy : PFI conventional therapy
4. Difference in outcome between patients have received recommended personalised therapy and those who did not (i. Proportion of patients without progression or death at 6 and 12 months between the two groups ii. Difference in progression-free and overall survival between the two groups)
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Timepoint [6]
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5 years
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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.
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Minimum age
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Years
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Maximum age
25
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/12/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/07/2030
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Actual
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Sample size
Target
3500
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Women's and Children's Hospital - Adelaide
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Recruitment hospital [2]
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Queensland Children's Hospital - Brisbane
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Recruitment hospital [3]
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Royal Hobart Hospital - Hobart
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Recruitment hospital [4]
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Monash Children's Hospital - Melbourne
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Recruitment hospital [5]
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Royal Children's Hospital - Melbourne
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Recruitment hospital [6]
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John Hunter Children's Hospital - Newcastle
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Recruitment hospital [7]
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Perth Children's Hospital - Perth
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Recruitment hospital [8]
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Sydney Children's Hospital, Randwick - Sydney
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Recruitment hospital [9]
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The Children's Hospital at Westmead - Sydney
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Recruitment postcode(s) [1]
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- Adelaide
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Recruitment postcode(s) [2]
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- Brisbane
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Recruitment postcode(s) [3]
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- Hobart
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Recruitment postcode(s) [4]
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- Melbourne
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Recruitment postcode(s) [5]
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- Newcastle
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Recruitment postcode(s) [6]
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- Perth
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Recruitment postcode(s) [7]
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- Sydney
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Recruitment outside Australia
Country [1]
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New Zealand
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State/province [1]
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Grafton
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Country [2]
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New Zealand
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State/province [2]
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Christchurch
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Funding & Sponsors
Primary sponsor type
Other
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Name
Australian & New Zealand Children's Haematology/Oncology Group
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Address
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Country
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Other collaborator category [1]
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0
Other
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Name [1]
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Children's Cancer Institute (CCI)
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Address [1]
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0
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Country [1]
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0
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Other collaborator category [2]
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Other
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Name [2]
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Minderoo Foundation
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Address [2]
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0
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Country [2]
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0
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Other collaborator category [3]
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Other
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Name [3]
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Medical Research Future Fund
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Address [3]
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Country [3]
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Ethics approval
Ethics application status
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Summary
Brief summary
To improve outcomes for childhood cancer patients through the implementation of precision medicine.
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Trial website
https://clinicaltrials.gov/study/NCT05504772
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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David Ziegler
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Address
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SCHN
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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National Study Coordinator
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Address
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Country
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Phone
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+61 2 9382 3102
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Fax
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Email
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[email protected]
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/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
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Prof
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Name
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David Ziegler
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Address
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Kids Cancer Centre Sydney Children’s Hospital Randwick, NSW 2031
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Country
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Australia
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Phone
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+61 (02) 9382 1730
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Fax
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+61 (02) 9382 1789
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Email
353
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[email protected]
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Contact person for public queries
Title
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Name
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KOALA NCC Team
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Address
354
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Kids Cancer Centre Sydney Children’s Hospital Randwick, NSW 2031
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Country
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Australia
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Phone
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+61 (02) 9382 1730
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Fax
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+61 (02) 9382 1789
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Email
354
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[email protected]
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Contact person for scientific queries
Title
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Prof
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Name
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David Ziegler
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Address
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Kids Cancer Centre Sydney Children’s Hospital Randwick, NSW 2031
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Country
355
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Australia
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Phone
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+61 (02) 9382 1730
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Fax
355
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+61 (02) 9382 1789
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Email
355
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[email protected]
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