PRecISion Medicine for Children With Cancer

Childhood Cancer Clinical Trial

— PRISM  

Official title:

A Multicenter 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03336931
• Other study ID #: PRISM
• Status: Active, not recruiting
• Start date: September 5, 2017
• Est. completion date: December 2032

Study information

• Verified date: April 2024
• Source: Sydney Children's Hospitals Network
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 550
• Est. completion date: December 2032
• Est. primary completion date: August 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

Inclusion criteria (all must be met)

1. Age = 21 years

2. Histologic diagnosis of high-risk malignancy defined as expected overall survival < 30% OR where standard therapy would result in unacceptable and severe morbidity

3. Appropriate tissue samples are available for analysis

4. Life expectancy > 6 weeks

5. Written informed consent

Related Conditions & MeSH terms

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

Intervention

Diagnostic Test:
• Molecular profiling and drug testing
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 Multi-disciplinary Tumour Board case discussion Recommendation of personalised therapy

Locations

Country	Name	City	State
Australia	Women's and Children's Hospital	Adelaide	South Australia
Australia	Queensland Children's Hospital	Brisbane	Queensland
Australia	Monash Children's Hospital	Melbourne	Victoria
Australia	Royal Children's Hospital	Melbourne	Victoria
Australia	John Hunter Children's Hospital	Newcastle	New South Wales
Australia	Perth Children's Hospital	Perth	Western Australia
Australia	Sydney Children's Hospital, Randwick	Sydney	New South Wales
Australia	The Children's Hospital at Westmead	Sydney	New South Wales

Sponsors (5)

Lead Sponsor	Collaborator
Sydney Children's Hospitals Network	Australian & New Zealand Children's Haematology/Oncology Group, Children's Cancer Institute Australia, Garvan Institute of Medical Research, German Cancer Research Center

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Impact of personalized therapy on progression-free survival	Time interval from enrollment until disease progression or death for patients who have received personalized therapy versus those who have not	Up to 5 years
Other	Impact of personalized therapy on overall survival	Time interval from enrollment until death for patients who have received personalized therapy versus those who have not	Up to 5 years
Primary	Personalized medicine recommendation	Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe	5 years
Secondary	Tumor samples with actionable molecular alterations	Proportion of tumor samples found to have actionable molecular alterations	5 years
Secondary	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	5 years
Secondary	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	5 years
Secondary	Reporting turnaround time	Number of weeks from enrollment to issuing a report to the treating clinician	5 years
Secondary	Patients receiving the recommended personalized therapy	Proportion of patients who subsequently receive the recommended personalized therapy	5 years
Secondary	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	5 years