A companion platform trial to test novel targeted agents based on the patient's tumor profile.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
90
Paxalisib starting at 21mg/m2 oral, daily, 28 day cycle, 13 cycles.
Opdualag, a fixed-dose combination of Nivolumab 480mg and Relatlimab 160mg, intravenous, on day 1, 28 day cycle, 26 cycles
Irinotecan starting at 50mg/m2/day, intravenous, on days 1-5, 28 day cycle, 13 cycles.
John Hunter Children's Hospital
Newcastle, New South Wales, Australia
RECRUITINGSydney Children's Hospital, Randwick
Sydney, New South Wales, Australia
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.
Time frame: 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.
Time frame: 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.
Time frame: 5 Years
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.
Time frame: 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.
Time frame: 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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Temozolomide starting at 150mg/m2/day, oral, on days 1-5, 28 day cycle, 13 cycles.
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
RECRUITINGQueensland Children's Hospital
Brisbane, Queensland, Australia
RECRUITINGWomen's and Children's Hospital
Adelaide, South Australia, Australia
RECRUITINGRoyal Hobart Hospital
Hobart, Tasmania, Australia
RECRUITINGMonash Children's Hospital
Melbourne, Victoria, Australia
RECRUITINGThe Royal Children's Hospital
Melbourne, Victoria, Australia
RECRUITINGPerth Children's Hospital
Perth, Western Australia, Australia
RECRUITINGStollery Children's Hospital
Edmonton, Canada
NOT_YET_RECRUITING...and 4 more locations
Time frame: 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.
Time frame: 5 Years