Precision Medicine for Every Child With Cancer
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Whole Genome Sequencing, RNA seq, DNA Methylation, Targeted Panel Sequencing.
- Who it may be relevant to
- Registry conditions: Childhood Cancer, Childhood Solid Tumor, Childhood Brain Tumor, Childhood Leukemia. Basic parameters: 0 years — 25 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia, New Zealand
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
To improve outcomes for childhood cancer patients through the implementation of precision medicine.
Detailed description
Through the pilot TARGET and national PRISM trials the feasibility and benefits of using comprehensive molecular profiling and preclinical drug testing in real time for high-risk (HR) patients has been demonstrated. However, the role of precision medicine, especially in facilitating diagnosis and risk stratification in non-HR childhood cancers has not been studied. Integrative tumor-germline whole genome sequencing (WGS) analysis has the potential to advance our understanding of cancer predisposition. In this study, the ZERO platform will be extended to all children with cancer in Australia and New Zealand, evaluating the benefits of precision medicine in different childhood cancer types and risk groups.
Interventions
- Genetic 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. - Genetic RNA seq
Results will be used for bioinformatics analysis for fusion transcripts and gene expression. - Genetic DNA Methylation
Genome-wide assessment of DNA methylation will be conducted on all samples where possible. - Genetic 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 - Genetic 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. - Genetic 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 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.
Primary outcome measures
- Utility of recommended personalized therapy for HR childhood cancer patients. [Time frame: 5 years]
- Utility of recommended personalized therapy for non-HR childhood cancer patients. [Time frame: 5 years]
Secondary outcome measures (6)
- Utility of pre-defined virtual molecular panel for non-HR childhood cancer patients. [Time frame: 5 years]
- Utility of comprehensive precision medicine for patients with rare tumors in childhood. [Time frame: 5 years]
- Utility of Molecular Tumour Board (MTB) recommendation tier system for HR childhood cancer patients. [Time frame: 5 years]
- Utility of preclinical testing in HR childhood cancer patients. [Time frame: 5 years]
- Clinical utility of germline WGS in patients with childhood cancers. [Time frame: 5 years]
- Treatment outcome in HR childhood cancer patients who have received recommended personalised therapy which are molecularly and/or preclinically directed. [Time frame: 5 years]
Eligibility criteria
Inclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Australia · 9 centers
- Women's and Children's Hospital — Adelaide
- Queensland Children's Hospital — Brisbane
- Royal Hobart Hospital — Hobart
- Monash Children's Hospital — Melbourne
- Royal Children's Hospital — Melbourne
- John Hunter Children's Hospital — Newcastle
- Perth Children's Hospital — Perth
- Sydney Children's Hospital, Randwick — Sydney
- … and 1 more center
New Zealand · 2 centers
- Starship Children's Hospital — Auckland
- Christchurch Hospital — Christchurch
Identifiers
NCT: NCT05504772 · ZERO2