PRecISion Medicine for Children With Cancer

Active, not recruiting

• Conditions: Childhood Cancer; Childhood Leukemia; Childhood Solid Tumor; Childhood Brain Tumor; Relapsed Cancer; Refractory Cancer
• Interventions: Diagnostic Test: Molecular profiling and drug testing

• Registration Number: NCT03336931
• Lead Sponsor: Sydney Children's Hospitals Network

Brief Summary

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.

Detailed Description

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 Timeline

• Study Start: 2017-09-05
• Study First Submitted: 2017-10-22
• Study First Submitted QC: 2017-11-05
• Study First Posted: 2017-11-08
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-22
• Last Update Posted: 2024-04-23
• Primary Completion: 2028-08
• Study Completion: 2032-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 550

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
High-risk childhood cancers	Molecular profiling and drug testing	Expected survival \< 30%

Primary Outcome Measures

Name	Time	Method
Personalized medicine recommendation	5 years	Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe

Secondary Outcome Measures

Name	Time	Method
Identification of potential treatment by in vitro or in vivo drug screening	5 years	Proportion of tumors for which a potential treatment option is identified by in vitro or in vivo drug screening
Patients receiving the recommended personalized therapy	5 years	Proportion of patients who subsequently receive the recommended personalized therapy
Tumor samples with actionable molecular alterations	5 years	Proportion of tumor samples found to have actionable molecular alterations
Successfully conducted in vitro high throughput drug screening and in vivo drug sensitivity testing	5 years	Proportion of tumours where in vitro high throughput drug screening and in vivo drug sensitivity testing can be successfully performed
Reporting turnaround time	5 years	Number of weeks from enrollment to issuing a report to the treating clinician
Barriers or reasons for patients not receiving the recommended personalized therapy	5 years	Description of the barriers or reasons for patients not receiving the recommended personalized therapy

Trial Locations

Locations (8)

The Children's Hospital at Westmead; 🇦🇺 Sydney, New South Wales, Australia

Women's and Children's Hospital; 🇦🇺 Adelaide, South Australia, Australia

Monash Children's Hospital; 🇦🇺 Melbourne, Victoria, Australia

Perth Children's Hospital; 🇦🇺 Perth, Western Australia, Australia

Queensland Children's Hospital; 🇦🇺 Brisbane, Queensland, Australia

Sydney Children's Hospital, Randwick; 🇦🇺 Sydney, New South Wales, Australia

John Hunter Children's Hospital; 🇦🇺 Newcastle, New South Wales, Australia

Royal Children's Hospital; 🇦🇺 Melbourne, Victoria, Australia