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| Name | Class |
|---|---|
| Children's Cancer Institute Australia | UNKNOWN |
| Australian & New Zealand Children's Haematology/Oncology Group | OTHER |
| Garvan Institute of Medical Research | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| High-risk childhood cancers | Expected survival < 30% |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Molecular profiling and drug testing | Diagnostic Test |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Tumor samples with actionable molecular alterations | Proportion of tumor samples found to have actionable molecular alterations | 5 years |
| Successfully conducted in vitro high throughput drug screening and in vivo drug sensitivity testing |
| Measure | Description | Time Frame |
|---|---|---|
| Impact of personalized therapy on progression-free survival | Time interval from enrollment until disease progression or death for patients who have received personalized therapy versus those who have not | Up to 5 years |
| Impact of personalized therapy on overall survival |
Inclusion criteria (all must be met)
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Paediatric patients who are being treated for high-risk cancer in Australia
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| Name | Affiliation | Role |
|---|---|---|
| A/Prof David Ziegler, MBBS | Sydney Children's Hospitals Network | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| John Hunter Children's Hospital | Newcastle | New South Wales | 2305 | Australia | ||
| Sydney Children's Hospital, Randwick |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42399266 | Derived | 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. | |
| 40209140 |
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| German Cancer Research Center |
| OTHER |
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Fresh, cryopreserved or frozen tumor, bone marrow or blood
|
Proportion of tumours where in vitro high throughput drug screening and in vivo drug sensitivity testing can be successfully performed
| 5 years |
| 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 | 5 years |
| Reporting turnaround time | Number of weeks from enrollment to issuing a report to the treating clinician | 5 years |
| Patients receiving the recommended personalized therapy | Proportion of patients who subsequently receive the recommended personalized therapy | 5 years |
| 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 | 5 years |
Time interval from enrollment until death for patients who have received personalized therapy versus those who have not |
| Up to 5 years |
| Sydney |
| New South Wales |
| 2031 |
| Australia |
| The Children's Hospital at Westmead | Sydney | New South Wales | 2145 | Australia |
| Queensland Children's Hospital | Brisbane | Queensland | 4101 | Australia |
| Women's and Children's Hospital | Adelaide | South Australia | 5006 | Australia |
| Royal Children's Hospital | Melbourne | Victoria | 3052 | Australia |
| Monash Children's Hospital | Melbourne | Victoria | 3168 | Australia |
| Perth Children's Hospital | Perth | Western Australia | 6008 | Australia |
| Derived |
| 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. |
| 38844796 | Derived | 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. |
| 36882456 | Derived | 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. |
| 32580982 | Derived | 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. |
| ID | Term |
|---|---|
| D009369 | Neoplasms |
| D012008 | Recurrence |
| ID | Term |
|---|---|
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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