Clinical trial NCT05504772: Precision Medicine for Every Child With Cancer
Official title: Precision Medicine for Every Child With Cancer
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Childhood Cancer
Childhood Solid Tumor
Childhood Brain Tumor
Childhood Leukemia
Refractory Cancer
Relapsed Cancer
Keywords: children
precision medicine
personalised medicine
molecular profiling
pediatric
paediatric
Lead sponsor: Australian & New Zealand Children's Haematology/Oncology Group
Sponsor class: OTHER
Collaborators: name: Children's Cancer Institute (CCI)
name: Minderoo Foundation
name: Medical Research Future Fund
First posted: 2022-08-17
Enrollment: 3500
Sex: ALL
Minimum age (years): 0
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients \<18 years of age with a diagnosis of tumor or cancer Patients aged 19 - 25 years with a diagnosis of a pediatric tumor or cancer

Brief summary: To improve outcomes for childhood cancer patients through the implementation of precision medicine.

Detailed description: Through the pilot TARGET and national PRISM trials the feasibility and benefits of using comprehensive molecular profiling and preclinical drug testing in real time for high-risk (HR) patients has been demonstrated. However, the role of precision medicine, especially in facilitating diagnosis and risk stratification in non-HR childhood cancers has not been studied. Integrative tumor-germline whole genome sequencing (WGS) analysis has the potential to advance our understanding of cancer predisposition. In this study, the ZERO platform will be extended to all children with cancer in Australia and New Zealand, evaluating the benefits of precision medicine in different childhood cancer types and risk groups.

Eligibility criteria: Inclusion Criteria:

1. Age \< 18 years Note: Individual patients aged 19 - 25 years old with a pediatric cancer, e.g., neuroblastoma, may be enrolled after discussion with, and at the discretion of, the Study Chair or their delegate.
2. Life expectancy \>6 weeks at time of enrolment
3. Consent i. Signed and dated informed consent for study enrolment from participant aged ≥ 18 years or from parent/guardian of participant aged \<18 years. ii. Separate signed and dated informed consent for understanding the role of germline testing and choice for the return of germline results.

Primary outcomes:
measure: Utility of recommended personalized therapy for HR childhood cancer patients.
timeFrame: 5 years
measure: Utility of recommended personalized therapy for non-HR childhood cancer patients.
timeFrame: 5 years
Secondary outcomes:
measure: Utility of pre-defined virtual molecular panel for non-HR childhood cancer patients.
timeFrame: 5 years
measure: Utility of comprehensive precision medicine for patients with rare tumors in childhood.
timeFrame: 5 years
measure: Utility of Molecular Tumour Board (MTB) recommendation tier system for HR childhood cancer patients.
timeFrame: 5 years
measure: Utility of preclinical testing in HR childhood cancer patients.
timeFrame: 5 years
measure: Clinical utility of germline WGS in patients with childhood cancers.
timeFrame: 5 years
measure: Treatment outcome in HR childhood cancer patients who have received recommended personalised therapy which are molecularly and/or preclinically directed.
timeFrame: 5 years
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Women's and Children's Hospital
city: Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Hobart
country: Australia
facility: Royal Hobart Hospital
city: Melbourne
country: Australia
facility: Monash Children's Hospital
city: Melbourne
country: Australia
facility: Royal Children's Hospital
city: Newcastle
country: Australia
facility: John Hunter Children's Hospital
city: Perth
country: Australia
facility: Perth Children's Hospital
city: Sydney
country: Australia
facility: Sydney Children's Hospital, Randwick
city: Sydney
country: Australia
facility: The Children's Hospital at Westmead
city: Auckland
country: New Zealand
facility: Starship Children's Hospital
state: Grafton
city: Christchurch
country: New Zealand
facility: Christchurch Hospital
Study officials:
affiliation: SCHN
name: David Ziegler
role: PRINCIPAL_INVESTIGATOR
References: