Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer
2 other identifiers
interventional
90
2 countries
14
Brief Summary
A companion platform trial to test novel targeted agents based on the patient's tumor profile.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Jul 2024
Longer than P75 for phase_1
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 12, 2023
CompletedFirst Posted
Study publicly available on registry
January 17, 2024
CompletedStudy Start
First participant enrolled
July 10, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2035
January 28, 2026
January 1, 2026
6.4 years
December 12, 2023
January 26, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
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 Outcomes (4)
Overall Clinical Benefit Rate (CBR) for each treatment arm
5 Years
Progression Free Survival (PFS) for each treatment arm.
5 Years
Incidence of treatment-emergent adverse events for each treatment arm.
5 Years
Maximum Concentration (Cmax) of molecularly-targeted agents for each treatment arm.
5 Years
Study Arms (2)
Arm A Paxalisib
EXPERIMENTALDrug: Irinotecan, Drug: Temozolomide, Drug: Paxalisib. Irinotecan starting at 50mg/m2/day, intravenous, on days 1-5, 28 day cycle, 13 cycles. Temozolomide starting at 150mg/m2/day, oral, on days 1-5, 28 day cycle, 13 cycles. Paxalisib starting at 21mg/m2 oral, daily, 28 day cycle, 13 cycles.
Arm C Opdualag
EXPERIMENTALDrug: Opdualag, a fixed dose combination of Nivolumab and Relatlimab Opdualag, a fixed-dose combination of Nivolumab 480mg and Relatlimab 160mg, intravenous, on day 1, 28 day cycle, 26 cycles.
Interventions
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.
Temozolomide starting at 150mg/m2/day, oral, on days 1-5, 28 day cycle, 13 cycles.
Eligibility Criteria
You may qualify if:
- Patients must be diagnosed with a solid tumor, CNS tumor or lymphoma that has progressed despite standard therapy, or for which no effective standard therapy exists.
- Patients must be enrolled on a precision medicine study (i.e. PROFYLE, ZERO or equivalent as agreed with Study Chair).
- Patients enrolled in a Phase I cohort must have either evaluable or measurable disease.
- Patients enrolled in a Phase II cohort must have measurable disease. Evaluable and measurable disease are defined by standard imaging criteria for the patient's tumor type.
- Disease evaluations, laboratory tests, and other clinical assessments that are considered standard of care may be undertaken at the patient's local oncology treatment centre with results transferred to study site for evaluation.
- Performance status: Karnofsky performance status (for patients \> 16 years of age) or Lansky play score (for patients ≤ 16 years of age) ≥ 50%.
- Life expectancy ≥ 6 weeks.
- Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy and must meet the following minimum duration from prior anticancer-directed therapy prior to enrolment.
- Adequate organ function.
- Able to comply with scheduled follow-up and with management of toxicity.
- Females of childbearing potential must have a negative serum or urine pregnancy test.
- Fertile males must agree to use adequate contraception during the study and following completion of treatment.
- Provide a signed and dated informed consent form.
You may not qualify if:
- Patients with symptomatic central nervous system (CNS) primary or metastatic tumours who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease. Patients on stable doses of corticosteroids for at least 7 days prior to receiving study drug may be included.
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhoea, or malabsorption syndrome) - only for arms that include orally administered therapeutic agents.
- Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias, e.g., ventricular, supraventricular, nodal arrhythmias, or conduction abnormality), unstable ischemia, congestive heart failure within 12 months of screening.
- Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
- Major surgery within 21 days of the first dose of investigational drug. Gastrostomy, ventriculo-peritoneal shunt, endoscopic ventriculostomy, tumour biopsy and insertion of central venous access devices are not considered major surgery, but for these procedures, a 48-hour interval must be maintained before the first dose of the investigational drug is administered.
- Known hypersensitivity to any study drug or component of the formulation.
- Pregnant or nursing (lactating) females.
- Any other concomitant serious medical condition or organ dysfunction that in the opinion of the investigator would either compromise patient safety or interfere with the evaluation of the safety of the investigational drug(s).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- The Hospital for Sick Childrencollaborator
- Medical Research Future Fundcollaborator
- C17 Councilcollaborator
- Australian & New Zealand Children's Haematology/Oncology Grouplead
- Kazia Therapeutics Limitedcollaborator
- Bristol-Myers Squibbcollaborator
- Stand Up To Cancercollaborator
Study Sites (14)
John Hunter Children's Hospital
Newcastle, New South Wales, Australia
Sydney Children's Hospital, Randwick
Sydney, New South Wales, Australia
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
Queensland Children's Hospital
Brisbane, Queensland, Australia
Women's and Children's Hospital
Adelaide, South Australia, Australia
Royal Hobart Hospital
Hobart, Tasmania, 7000, Australia
Monash Children's Hospital
Melbourne, Victoria, Australia
The Royal Children's Hospital
Melbourne, Victoria, Australia
Perth Children's Hospital
Perth, Western Australia, Australia
Stollery Children's Hospital
Edmonton, Canada
CHU Sainte Justine
Montreal, Canada
Children's Hospital of Eastern Ontario
Ottawa, Canada
The Hospital for Sick Children
Toronto, Canada
BC Children's Hospital
Vancouver, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
David Ziegler, Prof
Sydney Children's Hospital - Australian Study Chair
- STUDY CHAIR
Daniel Morgenstern, Dr
The Hospital for Sick Children - Canadian Study Chair
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 12, 2023
First Posted
January 17, 2024
Study Start
July 10, 2024
Primary Completion (Estimated)
December 1, 2030
Study Completion (Estimated)
December 1, 2035
Last Updated
January 28, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will share
Plan to Share IPD: Neither the complete nor any part of the results of the study carried out under this protocol, nor any of the information provided by the Sponsor for the purposes of performing the study, will be published or passed on to any third party without the consent of the Study Committee. The pseudonymized data will be shared for transparency reasons in the context of publications and after publication with other physicians and scientists (national and international academia) to promote and accelerate research on causes and treatment development of oncological diseases. Requests for access to pseudonymized patient data for other scientific purposes will be reviewed by the Study Committee. A positive statement of the respective ethic committee and a signed data protection commitment are requested. Results of scientific research based on the study data may be used for academic teaching, research and scientific publications or presentations at scientific meetings.