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PRecISion Medicine for Children With Cancer (PRISM)

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ClinicalTrials.gov Identifier: NCT03336931
Recruitment Status : Recruiting
First Posted : November 8, 2017
Last Update Posted : November 8, 2017
Sponsor:
Collaborators:
Children's Cancer Institute Australia
Australian and 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

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.

Condition or disease Intervention/treatment
Childhood Cancer Childhood Solid Tumor Childhood Brain Tumor Childhood Leukemia Refractory Cancer Relapsed Cancer Diagnostic Test: Molecular profiling and drug testing

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
Estimated Enrollment : 400 participants
Observational Model: Cohort
Time Perspective: Prospective
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
Actual Study Start Date : September 5, 2017
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2027

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
High-risk childhood cancers
Expected survival < 30%
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




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Tumor samples with actionable molecular alterations [ Time Frame: 5 years ]
    Proportion of tumor samples found to have actionable molecular alterations

  2. 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

  3. 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

  4. Reporting turnaround time [ Time Frame: 5 years ]
    Number of weeks from enrollment to issuing a report to the treating clinician

  5. Patients receiving the recommended personalized therapy [ Time Frame: 5 years ]
    Proportion of patients who subsequently receive the recommended personalized therapy

  6. 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


Other Outcome Measures:
  1. 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

  2. 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


Biospecimen Retention:   Samples With DNA
Fresh, cryopreserved or frozen tumor, bone marrow or blood


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Paediatric patients who are being treated for high-risk cancer in Australia
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03336931


Contacts
Contact: Clinical Trials Project Manager +61 2 9382 3122 SCHN-PRISMstudy@health.nsw.gov.au

Locations
Australia, New South Wales
John Hunter Children's Hospital Recruiting
Newcastle, New South Wales, Australia, 2305
Sydney Children's Hospital, Randwick Recruiting
Sydney, New South Wales, Australia, 2031
The Children's Hospital at Westmead Not yet recruiting
Sydney, New South Wales, Australia, 2145
Australia, Queensland
Lady Cilento Children's Hospital Recruiting
Brisbane, Queensland, Australia, 4101
Australia, South Australia
Women's and Children's Hospital Not yet recruiting
Adelaide, South Australia, Australia, 5006
Australia, Victoria
Royal Children's Hospital Not yet recruiting
Melbourne, Victoria, Australia, 3052
Monash Children's Hospital Not yet recruiting
Melbourne, Victoria, Australia, 3168
Australia, Western Australia
Perth Children's Hospital Recruiting
Perth, Western Australia, Australia, 6008
Sponsors and Collaborators
Sydney Children's Hospitals Network
Children's Cancer Institute Australia
Australian and 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

Responsible Party: Dr David Ziegler, Associate Professor, Sydney Children's Hospitals Network
ClinicalTrials.gov Identifier: NCT03336931     History of Changes
Other Study ID Numbers: PRISM
First Posted: November 8, 2017    Key Record Dates
Last Update Posted: November 8, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Dr David Ziegler, Sydney Children's Hospitals Network:
children
precision medicine
personalised medicine
high-risk cancer
refractory
recurrent
sequencing
patient derived xenograft
molecular profiling
paediatric