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Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer
A companion platform trial to test novel targeted agents based on the patient's tumor profile.
Study details:
Both Australia (Zero Childhood Cancer) and Canada (PROFYLE) have developed precision oncology programs for the pediatric population through which samples from childhood/adolescent cancers undergo in depth genetic profiling. OPTIMISE is a companion platform trial, which will link patients to novel targeted agents based on their tumor profile. The trial will have multiple basket arms based on the most common genetically altered pathways the investigators have identified in these childhood cancers.
Each arm of the trial will be histopathology agnostic and test a rational, novel combination therapy, to maximise potential clinical benefit.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 0 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2024-07-10
Primary completion: 2030-12-01
Study completion finish: 2035-12-01
Study type
TREATMENT
Phase
PHASE1
PHASE2
Trial ID
NCT06208657
Intervention or treatment
DRUG: Paxalisib, Irinotecan, Temozolomide
DRUG: Pimasertib
Conditions
- • Childhood Cancer
- • Childhood Solid Tumor
- • Childhood Brain Tumor
- • Recurrent Cancer
- • Refractory Cancer
Find a site
Closest Location:
The Children's Hospital at Westmead
Research sites nearby
Select from list below to view details:
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
John Hunter Children's Hospital
Newcastle, New South Wales, Australia
Sydney Children's Hospital, Randwick
Sydney, New South Wales, Australia
Queensland Children's Hospital
Brisbane, Queensland, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Arm A Paxalisib
| DRUG: Paxalisib, Irinotecan, Temozolomide
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Number of participants treated with molecularly-targeted agents in each treatment arm. | Number of CAYA participants (children, adolescents and young adults) with advanced solid tumours (including CNS tumors and non-Hodgkin lymphomas) where molecular sequencing data was used to allocate treatment arms of molecularly-targeted agents. | 5 Years |
Recommended phase II dose for each treatment arm | Recommended phase II dose of a novel single agent or combination treatment in CAYA participants, determined by dose-limiting toxicities reported as per CTCAE V5.0. | 3 Years |
Objective Response Rate (ORR) for each treatment arm. | ORR defined as complete response and partial response, as measured by RECIST, RAPNO, INRC or RECIL in CAYA participants treated with molecularly-targeted agents. | 5 Years |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Overall Clinical Benefit Rate (CBR) for each treatment arm | CBR defined as complete response and partial response and stable disease, as measured by RECIST, RAPNO, INRC or RECIL in CAYA participants treated with molecularly-targeted agents. | 5 Years |
Progression Free Survival (PFS) for each treatment arm. | PFS in CAYA participants from initiation of treatment with molecularly-targeted agents to the occurrence of disease progression, as measured by RECIST, RAPNO, INRC or RECIL, or death. | 5 Years |
Incidence of treatment-emergent adverse events for each treatment arm. | Safety and tolerability of molecularly-targeted agents as measured by incidence of treatment-emergent adverse events reported as per CTCAE V5.0 in CAYA participants. | 5 Years |
Maximum Concentration (Cmax) of molecularly-targeted agents for each treatment arm. | Cmax in plasma after the first dose of molecularly-targeted agents in CAYA participants. | 5 Years |
Frequently Asked Questions
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