NCT05504772

Precision Medicine for Every Child With Cancer

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 3,500 (estimated)
• Sponsor: Australian & New Zealand Children's Haematology/Oncology Group · Academic / Other
• Sex: All
• Age: 0 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Childhood Cancer
• Childhood Solid Tumor
• Childhood Brain Tumor
• Childhood Leukemia
• Refractory Cancer
• Relapsed Cancer

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2022-12-16
• Primary completion: 2025-07-01
• Completion: 2030-07-01
• First posted: 2022-08-17
• Last updated: 2024-07-17

Locations

11 sites across 2 countries: Australia, New Zealand