AzTrials
AzTrials

Childhood CancerPRISMNCT03336931

PRecISion Medicine for Children With Cancer

An observational study of Childhood Cancer and Childhood Solid Tumor, run by Sydney Children's Hospitals Network at 8 sites in Australia.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

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. Read more about this study

Who is it for?
Ages Up to 21 years
What is being tested?
Molecular profiling and drug testing
How many people
550 participants
Where
8 sitesAustralia
Study dates
5 Sep 2017to Dec 2032
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Study population: Paediatric patients who are being treated for high-risk cancer in Australia

Who may be able to join

  • Age ≤ 21 years
  • Histologic diagnosis of high-risk malignancy defined as expected overall survival < 30% OR where standard therapy would result in unacceptable and severe morbidity
  • Appropriate tissue samples are available for analysis
  • Life expectancy > 6 weeks
  • Written informed consent

Who cannot join

None listed.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

Australia8 sites

  • Women's and Children's Hospital

    Adelaide, South Australia, 5006

  • Queensland Children's Hospital

    Brisbane, Queensland, 4101

  • Royal Children's Hospital

    Melbourne, Victoria, 3052

  • Monash Children's Hospital

    Melbourne, Victoria, 3168

  • John Hunter Children's Hospital

    Newcastle, New South Wales, 2305

  • Perth Children's Hospital

    Perth, Western Australia, 6008

  • Sydney Children's Hospital, Randwick

    Sydney, New South Wales, 2031

  • The Children's Hospital at Westmead

    Sydney, New South Wales, 2145

Other trials in Australia

About this study

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

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.

Read the 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.

Being tested
Molecular profiling and drug testing
Keywords
children, precision medicine, personalised medicine, high-risk cancer, refractory, recurrent, sequencing, patient derived xenograft, molecular profiling, paediatric
Medical subject headings
Neoplasms, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Molecular profiling and drug testingDiagnostic test

    1. Laboratory analysis including:
    2. 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
    3. Multi-disciplinary Tumour Board case discussion
    4. Recommendation of personalised therapy
Study groups in NCT03336931
Study groupRoleWhat they receive
High-risk childhood cancersExpected survival < 30%
Receives: Diagnostic Test: Molecular profiling and drug testing

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Personalized medicine recommendation

    Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe

    Measured: 5 years

Secondary outcomes (6)

  1. Tumor samples with actionable molecular alterations

    Proportion of tumor samples found to have actionable molecular alterations

    Measured: 5 years

  2. 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

    Measured: 5 years

  3. 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

    Measured: 5 years

  4. Reporting turnaround time

    Number of weeks from enrollment to issuing a report to the treating clinician

    Measured: 5 years

  5. Patients receiving the recommended personalized therapy

    Proportion of patients who subsequently receive the recommended personalized therapy

    Measured: 5 years

  6. 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

    Measured: 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Nov 2017
  2. Study start5 Sep 2017
  3. Primary completionAug 2028 (estimated)
  4. Study completionDec 2032 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
550 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03336931
Sponsor's protocol ID
PRISM
Acronym
PRISM
Status last verified
Apr 2024
First submitted
22 Oct 2017
Last updated
23 Apr 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Hetherington K, Hunter JD, Donoghoe MW, McGillycuddy M, McGill BC, Robertson EG, Tyrrell V, Lau LMS, Marron JM, Tucker KM, Marshall GM, Vetsch J, Haber M, Malkin D, Mateos MK, O'Brien TA, Ziegler DS, Wakefield CE. Family experiences of receiving treatment recommendations in a precision medicine trial for poor-prognosis childhood cancer. NPJ Genom Med. 2026 Jul 3. doi: 10.1038/s41525-026-00591-y. Online ahead of print.

    PubMed 42399266

  • Robertson EG, Hetherington K, Hunter JD, McGillycuddy M, Venkatesha V, Lau LMS, Khuong-Quang DA, Ziegler DS, Wakefield CE. Whatever It Takes: Parents' Perspectives of Patient-Derived Xenograft Mouse Models for Poor Prognosis Childhood Cancer. JCO Precis Oncol. 2025 Apr;9:e2400840. doi: 10.1200/PO-24-00840. Epub 2025 Apr 10.

    PubMed 40209140

  • Robertson EG, Hetherington K, Daly R, Donoghoe MW, Handelsman N, Ziegler DS, Wakefield CE. The feasibility and acceptability of collecting psychosocial outcome measures embedded within a precision medicine trial for childhood cancer. Cancer Med. 2024 Jun;13(12):e7339. doi: 10.1002/cam4.7339.

    PubMed 38898768

  • Lau LMS, Khuong-Quang DA, Mayoh C, Wong M, Barahona P, Ajuyah P, Senapati A, Nagabushan S, Sherstyuk A, Altekoester AK, Fuentes-Bolanos NA, Yeung V, Sullivan A, Omer N, Diamond Y, Jessop S, Battaglia L, Zhukova N, Cui L, Lin A, Gifford AJ, Fleuren EDG, Dalla-Pozza L, Moore AS, Khaw SL, Eisenstat DD, Gottardo NG, Wood PJ, Tapp H, Alvaro F, McCowage G, Nicholls W, Hansford JR, Manoharan N, Kotecha RS, Mateos MK, Lock RB, Tyrrell V, Haber M, Trahair TN, Cowley MJ, Ekert PG, Marshall GM, Ziegler DS. Precision-guided treatment in high-risk pediatric cancers. Nat Med. 2024 Jul;30(7):1913-1922. doi: 10.1038/s41591-024-03044-0. Epub 2024 Jun 6.

    PubMed 38844796

  • Ajuyah P, Mayoh C, Lau LMS, Barahona P, Wong M, Chambers H, Valdes-Mora F, Senapati A, Gifford AJ, D'Arcy C, Hansford JR, Manoharan N, Nicholls W, Williams MM, Wood PJ, Cowley MJ, Tyrrell V, Haber M, Ekert PG, Ziegler DS, Khuong-Quang DA. Histone H3-wild type diffuse midline gliomas with H3K27me3 loss are a distinct entity with exclusive EGFR or ACVR1 mutation and differential methylation of homeobox genes. Sci Rep. 2023 Mar 7;13(1):3775. doi: 10.1038/s41598-023-30395-4.

    PubMed 36882456

  • 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.

    PubMed 32580982

Questions about this trial

What is this clinical trial studying?

An observational study of Childhood Cancer and Childhood Solid Tumor, run by Sydney Children's Hospitals Network at 8 sites in Australia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 23 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Personalized medicine recommendation (time frame: 5 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03336931, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in