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PRecISion Medicine for Children With Cancer (PRISM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03336931
Recruitment Status : Recruiting
First Posted : November 8, 2017
Last Update Posted : September 15, 2022
Sponsor:
Collaborators:
Children's Cancer Institute Australia
Australian & New Zealand Children's Haematology/Oncology Group
Garvan Institute of Medical Research
German Cancer Research Center
Information provided by (Responsible Party):
Dr David Ziegler, Sydney Children's Hospitals Network

Tracking Information
First Submitted Date October 22, 2017
First Posted Date November 8, 2017
Last Update Posted Date September 15, 2022
Actual Study Start Date September 5, 2017
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: November 5, 2017)
Personalized medicine recommendation [ Time Frame: 5 years ]
Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: November 5, 2017)
  • Tumor samples with actionable molecular alterations [ Time Frame: 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 [ Time Frame: 5 years ]
    Proportion of tumours where in vitro high throughput drug screening and in vivo drug sensitivity testing can be successfully performed
  • Identification of potential treatment by in vitro or in vivo drug screening [ Time Frame: 5 years ]
    Proportion of tumors for which a potential treatment option is identified by in vitro or in vivo drug screening
  • Reporting turnaround time [ Time Frame: 5 years ]
    Number of weeks from enrollment to issuing a report to the treating clinician
  • Patients receiving the recommended personalized therapy [ Time Frame: 5 years ]
    Proportion of patients who subsequently receive the recommended personalized therapy
  • Barriers or reasons for patients not receiving the recommended personalized therapy [ Time Frame: 5 years ]
    Description of the barriers or reasons for patients not receiving the recommended personalized therapy
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures
 (submitted: November 5, 2017)
  • Impact of personalized therapy on progression-free survival [ Time Frame: Up to 5 years ]
    Time interval from enrollment until disease progression or death for patients who have received personalized therapy versus those who have not
  • Impact of personalized therapy on overall survival [ Time Frame: Up to 5 years ]
    Time interval from enrollment until death for patients who have received personalized therapy versus those who have not
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title PRecISion Medicine for Children With Cancer
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
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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Fresh, cryopreserved or frozen tumor, bone marrow or blood
Sampling Method Non-Probability Sample
Study Population Paediatric patients who are being treated for high-risk cancer in Australia
Condition
  • Childhood Cancer
  • Childhood Solid Tumor
  • Childhood Brain Tumor
  • Childhood Leukemia
  • Refractory Cancer
  • Relapsed Cancer
Intervention Diagnostic Test: Molecular profiling and drug testing
  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
Study Groups/Cohorts High-risk childhood cancers
Expected survival < 30%
Intervention: Diagnostic Test: Molecular profiling and drug testing
Publications * Rapport F, Smith J, O'Brien TA, Tyrrell VJ, Mould EV, Long JC, Gul H, Braithwaite J. Development of an implementation and evaluation strategy for the Australian 'Zero Childhood Cancer' (Zero) Program: a study protocol. BMJ Open. 2020 Jun 23;10(6):e034522. doi: 10.1136/bmjopen-2019-034522.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: December 5, 2021)
550
Original Estimated Enrollment
 (submitted: November 5, 2017)
400
Estimated Study Completion Date December 2027
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Clinical Trials Project Manager +61 2 9382 3122 SCHN-PRISMstudy@health.nsw.gov.au
Listed Location Countries Australia
Removed Location Countries  
 
Administrative Information
NCT Number NCT03336931
Other Study ID Numbers PRISM
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Current Responsible Party Dr David Ziegler, Sydney Children's Hospitals Network
Original Responsible Party Dr David Ziegler, Sydney Children's Hospitals Network, Associate Professor
Current Study Sponsor Sydney Children's Hospitals Network
Original Study Sponsor Same as current
Collaborators
  • Children's Cancer Institute Australia
  • Australian & New Zealand Children's Haematology/Oncology Group
  • Garvan Institute of Medical Research
  • German Cancer Research Center
Investigators
Principal Investigator: A/Prof David Ziegler, MBBS Sydney Children's Hospitals Network
PRS Account Sydney Children's Hospitals Network
Verification Date September 2022