Precision Medicine for Every Child With Cancer
ZERO2
1 other identifier
observational
3,500
2 countries
11
Brief Summary
To improve outcomes for childhood cancer patients through the implementation of precision medicine.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Dec 2022
Longer than P75 for all trials
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 30, 2022
CompletedFirst Posted
Study publicly available on registry
August 17, 2022
CompletedStudy Start
First participant enrolled
December 16, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2030
ExpectedJuly 17, 2024
July 1, 2024
2.5 years
January 30, 2022
July 16, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Utility of recommended personalized therapy for HR childhood cancer patients.
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
5 years
Utility of recommended personalized therapy for non-HR childhood cancer patients.
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.
5 years
Secondary Outcomes (6)
Utility of pre-defined virtual molecular panel for non-HR childhood cancer patients.
5 years
Utility of comprehensive precision medicine for patients with rare tumors in childhood.
5 years
Utility of Molecular Tumour Board (MTB) recommendation tier system for HR childhood cancer patients.
5 years
Utility of preclinical testing in HR childhood cancer patients.
5 years
Clinical utility of germline WGS in patients with childhood cancers.
5 years
- +1 more secondary outcomes
Study Arms (13)
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.
Interventions
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.
Results will be used for bioinformatics analysis for fusion transcripts and gene expression.
Genome-wide assessment of DNA methylation will be conducted on all samples where possible.
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
High throughput drug screening will be attempted for tumors from Cohort 1 (high-risk cancers with survival \<30%) and selected tumor types.
In vivo drug testing in patient derived xenograft (PDX) will be attempted for tumors from Cohort 1 (high-risk cancers) and selected tumor types.
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.
Eligibility Criteria
Patients \<18 years of age with a diagnosis of tumor or cancer Patients aged 19 - 25 years with a diagnosis of a pediatric tumor or cancer
You may qualify if:
- 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.
- Life expectancy \>6 weeks at time of enrolment
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Australian & New Zealand Children's Haematology/Oncology Grouplead
- Children's Cancer Institute (CCI)collaborator
- Minderoo Foundationcollaborator
- Medical Research Future Fundcollaborator
Study Sites (11)
Women's and Children's Hospital
Adelaide, Australia
Queensland Children's Hospital
Brisbane, Australia
Royal Hobart Hospital
Hobart, Australia
Monash Children's Hospital
Melbourne, Australia
Royal Children's Hospital
Melbourne, Australia
John Hunter Children's Hospital
Newcastle, Australia
Perth Children's Hospital
Perth, Australia
Sydney Children's Hospital, Randwick
Sydney, Australia
The Children's Hospital at Westmead
Sydney, Australia
Starship Children's Hospital
Auckland, Grafton, 1023, New Zealand
Christchurch Hospital
Christchurch, 8011, New Zealand
Biospecimen
Tumour, blood, bone marrow, cerebrospinal fluid (CSF), urine, skin biopsy, cytogenetic culture, extracted DNA and peripheral blood stem cells.
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
David Ziegler
SCHN
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 30, 2022
First Posted
August 17, 2022
Study Start
December 16, 2022
Primary Completion
July 1, 2025
Study Completion (Estimated)
July 1, 2030
Last Updated
July 17, 2024
Record last verified: 2024-07