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RecruitingNCT06208657Updated Jan 28, 2026

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

A Phase 1/2 interventional study of Paxalisib and Opdualag in Childhood Cancer, Childhood Solid Tumor and Childhood Brain Tumor, sponsored by Australian & New Zealand Children's Haematology/Oncology Group. Recruiting at 14 sites in 2 countries. Open to participants aged 0 Years to 21 Years. Per ClinicalTrials.gov, last updated 2026-01-28.

Sponsored by Australian & New Zealand Children's Haematology/Oncology Group · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Started Jul 2024; still recruiting 2 years 2 months later.
Phase
Phase 1/2
Study type
Interventional
Enrollment
90
Allocation
Non-randomized
Ages
0 Years to 21 Years
Sex
All
01

Study summary

A companion platform trial to test novel targeted agents based on the patient's tumor profile.

Read the detailed description

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.

02

Conditions studied

  • Childhood Cancer
  • Childhood Solid Tumor
  • Childhood Brain Tumor
  • Recurrent Cancer
  • Refractory Cancer

Keywords

  • children
  • basket trial
  • platform study
  • pediatric
  • solid tumor
  • paediatric
  • solid tumour
  • CNS tumor
  • CNS tumour
  • lymphoma
03

In context

Neoplasms

9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.

This study's planned enrollment of 90 is above the median of 50 across 7,253 interventional studies indexed under Neoplasms.

Browse Neoplasms studies →

Lead sponsor

Australian & New Zealand Children's Haematology/Oncology Group is the lead sponsor of 3 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
0 Years to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. 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.
  2. Age \<21 years at inclusion; patients 21 years and older may be included after approval by the Study Chair if they have a pediatric type recurrent/refractory malignancy.
  3. Patients must be enrolled on a precision medicine study (i.e. PROFYLE, ZERO or equivalent as agreed with Study Chair).
  4. Patients enrolled in a Phase I cohort must have either evaluable or measurable disease.
  5. 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.
  6. 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.
  7. Performance status: Karnofsky performance status (for patients > 16 years of age) or Lansky play score (for patients ≤ 16 years of age) ≥ 50%.
  8. Life expectancy ≥ 6 weeks.
  9. 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.
  10. Adequate organ function.
  11. Able to comply with scheduled follow-up and with management of toxicity.
  12. Females of childbearing potential must have a negative serum or urine pregnancy test.
  13. Fertile males must agree to use adequate contraception during the study and following completion of treatment.
  14. Provide a signed and dated informed consent form.

Exclusion criteria

Exclusion Criteria:

  1. 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.
  2. 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.
  3. 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.
  4. Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
  5. 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.
  6. Known hypersensitivity to any study drug or component of the formulation.
  7. Pregnant or nursing (lactating) females.
  8. 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).
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
90 participants (estimated)

Study arms

  • Experimental
    Arm A Paxalisib

    Drug: 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.

    Drug: Paxalisib · Drug: Irinotecan (drug) · Drug: Temozolomide (TMZ)

  • Experimental
    Arm C Opdualag

    Drug: 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.

    Drug: Opdualag

Interventions

  • DrugPaxalisib

    Paxalisib starting at 21mg/m2 oral, daily, 28 day cycle, 13 cycles.

  • DrugOpdualag

    Opdualag, a fixed-dose combination of Nivolumab 480mg and Relatlimab 160mg, intravenous, on day 1, 28 day cycle, 26 cycles

  • DrugIrinotecan (drug)

    Irinotecan starting at 50mg/m2/day, intravenous, on days 1-5, 28 day cycle, 13 cycles.

  • DrugTemozolomide (TMZ)

    Temozolomide starting at 150mg/m2/day, oral, on days 1-5, 28 day cycle, 13 cycles.

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

  3. 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

Secondary outcomes

  1. 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

  2. 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

  3. 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.

    Time frame: 5 Years

  4. 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

07

Study locations

12 of 14 sites recruiting
08

References and documents

Individual participant data

Plan to share: Yes — 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.

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 28, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06208657
Lead sponsor
Australian & New Zealand Children's Haematology/Oncology Group
Collaborators
The Hospital for Sick Children, Medical Research Future Fund, Kazia Therapeutics Limited, C17 Council, Bristol-Myers Squibb, Stand Up To Cancer
Responsible party
Sponsor
First posted
Jan 17, 2024
Start date
Jul 10, 2024
Primary completion
Dec 2030 (estimated)
Completion
Dec 2035 (estimated)
Last update
Jan 28, 2026

Study contacts

International Study Coordinator
Contact
SCHN-OPTIMISE@health.nsw.gov.au
+61 2 9382 1730
International Study Manager
Contact
SCHN-OPTIMISE@health.nsw.gov.au
David Ziegler, Prof
study chair · Sydney Children's Hospital - Australian Study Chair
Daniel Morgenstern, Dr
study chair · The Hospital for Sick Children - Canadian Study Chair

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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