NCT03336931

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 Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
550

participants targeted

Target at P75+ for all trials

Timeline
80mo left

Started Sep 2017

Longer than P75 for all trials

Geographic Reach
1 country

8 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress57%
Sep 2017Dec 2032

Study Start

First participant enrolled

September 5, 2017

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

October 22, 2017

Completed
17 days until next milestone

First Posted

Study publicly available on registry

November 8, 2017

Completed
10.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2028

Expected
4.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2032

Last Updated

April 23, 2024

Status Verified

April 1, 2024

Enrollment Period

10.9 years

First QC Date

October 22, 2017

Last Update Submit

April 22, 2024

Conditions

Childhood CancerChildhood Solid TumorChildhood Brain TumorChildhood LeukemiaRefractory CancerRelapsed Cancer

Keywords

childrenprecision medicinepersonalised medicinehigh-risk cancerrefractoryrecurrentsequencingpatient derived xenograftmolecular profilingpaediatric

Outcome Measures

Primary Outcomes (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

    5 years

Secondary Outcomes (6)

  • Tumor samples with actionable molecular alterations

    5 years

  • Successfully conducted in vitro high throughput drug screening and in vivo drug sensitivity testing

    5 years

  • Identification of potential treatment by in vitro or in vivo drug screening

    5 years

  • Reporting turnaround time

    5 years

  • Patients receiving the recommended personalized therapy

    5 years

  • +1 more secondary outcomes

Other Outcomes (2)

  • Impact of personalized therapy on progression-free survival

    Up to 5 years

  • Impact of personalized therapy on overall survival

    Up to 5 years

Study Arms (1)

High-risk childhood cancers

Expected survival \< 30%

Diagnostic Test: Molecular profiling and drug testing

Interventions

Molecular profiling and drug testingDIAGNOSTIC_TEST

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

High-risk childhood cancers

Eligibility Criteria

AgeUp to 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

John Hunter Children's Hospital

Newcastle, New South Wales, 2305, Australia

Location

Sydney Children's Hospital, Randwick

Sydney, New South Wales, 2031, Australia

Location

The Children's Hospital at Westmead

Sydney, New South Wales, 2145, Australia

Location

Queensland Children's Hospital

Brisbane, Queensland, 4101, Australia

Location

Women's and Children's Hospital

Adelaide, South Australia, 5006, Australia

Location

Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

Monash Children's Hospital

Melbourne, Victoria, 3168, Australia

Location

Perth Children's Hospital

Perth, Western Australia, 6008, Australia

Location

Related Publications (4)

  • 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.

    PMID: 40209140DERIVED

  • 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.

    PMID: 38844796DERIVED

  • 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.

    PMID: 36882456DERIVED

  • 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.

    PMID: 32580982DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Fresh, cryopreserved or frozen tumor, bone marrow or blood

MeSH Terms

Conditions

NeoplasmsRecurrence

Condition Hierarchy (Ancestors)

Disease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • A/Prof David Ziegler, MBBS

    Sydney Children's Hospitals Network

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

October 22, 2017

First Posted

November 8, 2017

Study Start

September 5, 2017

Primary Completion (Estimated)

August 1, 2028

Study Completion (Estimated)

December 1, 2032

Last Updated

April 23, 2024

Record last verified: 2024-04

Locations

AU(8)