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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03336931




Registration number
NCT03336931
Ethics application status
Date submitted
22/10/2017
Date registered
8/11/2017
Date last updated
20/08/2020

Titles & IDs
Public title
PRecISion Medicine for Children With Cancer
Scientific 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
Secondary ID [1] 0 0
PRISM
Universal Trial Number (UTN)
Trial acronym
PRISM
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Childhood Cancer 0 0
Childhood Solid Tumor 0 0
Childhood Brain Tumor 0 0
Childhood Leukemia 0 0
Refractory Cancer 0 0
Relapsed Cancer 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Diagnosis / Prognosis - Molecular profiling and drug testing

High-risk childhood cancers - Expected survival < 30%


Diagnosis / Prognosis: 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

Intervention code [1] 0 0
Diagnosis / Prognosis
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Personalized medicine recommendation - Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe
Timepoint [1] 0 0
5 years
Secondary outcome [1] 0 0
Tumor samples with actionable molecular alterations - Proportion of tumor samples found to have actionable molecular alterations
Timepoint [1] 0 0
5 years
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
5 years
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
5 years
Secondary outcome [4] 0 0
Reporting turnaround time - Number of weeks from enrollment to issuing a report to the treating clinician
Timepoint [4] 0 0
5 years
Secondary outcome [5] 0 0
Patients receiving the recommended personalized therapy - Proportion of patients who subsequently receive the recommended personalized therapy
Timepoint [5] 0 0
5 years
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
5 years

Eligibility
Key inclusion criteria
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
Minimum age
No limit
Maximum age
21 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
John Hunter Children's Hospital - Newcastle
Recruitment hospital [2] 0 0
Sydney Children's Hospital, Randwick - Sydney
Recruitment hospital [3] 0 0
The Children's Hospital at Westmead - Sydney
Recruitment hospital [4] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [5] 0 0
Women's and Children's Hospital - Adelaide
Recruitment hospital [6] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [7] 0 0
Monash Children's Hospital - Melbourne
Recruitment hospital [8] 0 0
Perth Children's Hospital - Perth
Recruitment postcode(s) [1] 0 0
2305 - Newcastle
Recruitment postcode(s) [2] 0 0
2031 - Sydney
Recruitment postcode(s) [3] 0 0
2145 - Sydney
Recruitment postcode(s) [4] 0 0
4101 - Brisbane
Recruitment postcode(s) [5] 0 0
5006 - Adelaide
Recruitment postcode(s) [6] 0 0
3052 - Melbourne
Recruitment postcode(s) [7] 0 0
3168 - Melbourne
Recruitment postcode(s) [8] 0 0
6008 - Perth

Funding & Sponsors
Primary sponsor type
Other
Name
Sydney Children's Hospitals Network
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Children's Cancer Institute Australia
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Australian and New Zealand Children's Haematology/Oncology Group
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Garvan Institute of Medical Research
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
German Cancer Research Center
Address [4] 0 0
Country [4] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03336931
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
A/Prof David Ziegler, MBBS
Address 0 0
Sydney Children's Hospitals Network
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trials Project Manager
Address 0 0
Country 0 0
Phone 0 0
+61 2 9382 3122
Fax 0 0
Email 0 0
SCHN-PRISMstudy@health.nsw.gov.au
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03336931