This is a multicentre prospective study of the feasibility and clinical value of a diagnostic service for identifying therapeutic targets and recommending personalised treatment for children and adolescents with high-risk cancer.
This is a multicentre study conducted under the Zero Childhood Cancer Program. The study will be enrolling patients under the age of 21 with high-risk cancer over 3 years from cancer centres in Australia. Patient's cancer cells will be tested for genetic abnormalities (mutations) and undergoing drug testing in highly specialised laboratories. A Multidisciplinary Tumour Board comprising of oncologists, clinical geneticists and scientists will then discuss the results of each case and determine whether a personalised medicine recommendation can be made. A report describing the results and Tumour Board recommendation (if any) will be provided to the patient's treating doctor. It is always at the discretion of the treating doctor whether to alter the patient's management based on the information arising from this research project.
Study Type
OBSERVATIONAL
Enrollment
550
1. Laboratory analysis including: A. Tumour molecular profiling: targeted whole exon variant analysis, whole genome (DNA) and transcriptome (RNA) sequencing, methylation analysis, proteomics analysis, immunohistochemistry B. In vitro high-throughput drug sensitivity testing C. In vivo drug testing using patient-derived xenograft (PDX) models D. Liquid biopsies 2. Multi-disciplinary Tumour Board case discussion 3. Recommendation of personalised therapy
John Hunter Children's Hospital
Newcastle, New South Wales, Australia
Sydney Children's Hospital, Randwick
Sydney, New South Wales, Australia
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
Queensland Children's Hospital
Brisbane, Queensland, Australia
Personalized medicine recommendation
Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe
Time frame: 5 years
Tumor samples with actionable molecular alterations
Proportion of tumor samples found to have actionable molecular alterations
Time frame: 5 years
Successfully conducted in vitro high throughput drug screening and in vivo drug sensitivity testing
Proportion of tumours where in vitro high throughput drug screening and in vivo drug sensitivity testing can be successfully performed
Time frame: 5 years
Identification of potential treatment by in vitro or in vivo drug screening
Proportion of tumors for which a potential treatment option is identified by in vitro or in vivo drug screening
Time frame: 5 years
Reporting turnaround time
Number of weeks from enrollment to issuing a report to the treating clinician
Time frame: 5 years
Patients receiving the recommended personalized therapy
Proportion of patients who subsequently receive the recommended personalized therapy
Time frame: 5 years
Barriers or reasons for patients not receiving the recommended personalized therapy
Description of the barriers or reasons for patients not receiving the recommended personalized therapy
Time frame: 5 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Women's and Children's Hospital
Adelaide, South Australia, Australia
Royal Children's Hospital
Melbourne, Victoria, Australia
Monash Children's Hospital
Melbourne, Victoria, Australia
Perth Children's Hospital
Perth, Western Australia, Australia