Clinical trial NCT03336931: PRecISion Medicine for Children With Cancer
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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Childhood Cancer
Childhood Solid Tumor
Childhood Brain Tumor
Childhood Leukemia
Refractory Cancer
Relapsed Cancer
Keywords: children
precision medicine
personalised medicine
high-risk cancer
refractory
recurrent
sequencing
patient derived xenograft
molecular profiling
paediatric
Lead sponsor: Sydney Children's Hospitals Network
Sponsor class: OTHER
Collaborators: name: Children's Cancer Institute Australia
name: Australian & New Zealand Children's Haematology/Oncology Group
name: Garvan Institute of Medical Research
name: German Cancer Research Center
First posted: 2017-11-08
Enrollment: 550
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Paediatric patients who are being treated for high-risk cancer in Australia

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Personalized medicine recommendation
timeFrame: 5 years
Secondary outcomes:
measure: Tumor samples with actionable molecular alterations
timeFrame: 5 years
measure: Successfully conducted in vitro high throughput drug screening and in vivo drug sensitivity testing
timeFrame: 5 years
measure: Identification of potential treatment by in vitro or in vivo drug screening
timeFrame: 5 years
measure: Reporting turnaround time
timeFrame: 5 years
measure: Patients receiving the recommended personalized therapy
timeFrame: 5 years
measure: Barriers or reasons for patients not receiving the recommended personalized therapy
timeFrame: 5 years
Other outcomes:
measure: Impact of personalized therapy on progression-free survival
timeFrame: Up to 5 years
measure: Impact of personalized therapy on overall survival
timeFrame: Up to 5 years
Locations:
city: Newcastle
country: Australia
facility: John Hunter Children's Hospital
state: New South Wales
city: Sydney
country: Australia
facility: Sydney Children's Hospital, Randwick
state: New South Wales
city: Sydney
country: Australia
facility: The Children's Hospital at Westmead
state: New South Wales
city: Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Adelaide
country: Australia
facility: Women's and Children's Hospital
state: South Australia
city: Melbourne
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Melbourne
country: Australia
facility: Monash Children's Hospital
state: Victoria
city: Perth
country: Australia
facility: Perth Children's Hospital
state: Western Australia
Study officials:
affiliation: Sydney Children's Hospitals Network
name: A/Prof David Ziegler, MBBS
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.