Clinical trial · Observational
PRecISion Medicine for Children With Cancer
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
NCT03336931CI-TRIAL-00076366PRISMactive not recruitingClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (6)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Childhood Brain Tumor | Childhood Brain Neoplasm | ALIAS | 0.90 |
| Childhood Cancer | Childhood Malignant Neoplasm | ALIAS | 0.90 |
| Childhood Leukemia | Childhood Leukemia | ONTOLOGY_EXACT | 0.98 |
| Childhood Solid Tumor | Childhood Solid Neoplasm | ALIAS | 0.90 |
| Refractory Cancer | Malignant Neoplasm | CURATED_BROADER | 0.78 |
| Relapsed Cancer | Malignant Neoplasm | CURATED_BROADER | 0.78 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Molecular profiling and drug testing | Diagnostic Test | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- label
- High-risk childhood cancers
- description
- Expected survival \< 30%
- interventionNames
- Diagnostic Test: Molecular profiling and drug testing
Primary outcomes (1)
- measure
- Personalized medicine recommendation
- timeFrame
- 5 years
- description
- Proportion of patients for whom personalized medicine recommendation can be made using a comprehensive diagnostic platform within a clinically relevant timeframe
Secondary outcomes (6)
- measure
- Tumor samples with actionable molecular alterations
- timeFrame
- 5 years
- description
- Proportion of tumor samples found to have actionable molecular alterations
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
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
References
Publications (6)
- DERIVEDHetherington 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. PMID 42399266
- DERIVEDRobertson 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 40209140
- DERIVEDRobertson 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. PMID 38898768
- DERIVEDLau 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 38844796
- DERIVEDAjuyah 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.