Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

NCT ID: NCT06208657

Last Updated: 2026-01-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

90 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-10

Study Completion Date

2035-12-31

Brief Summary

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

Detailed Description

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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.

Conditions

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

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Paxalisib

Intervention Type DRUG

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

Irinotecan (drug)

Intervention Type DRUG

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

Temozolomide (TMZ)

Intervention Type DRUG

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

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.

Group Type EXPERIMENTAL

Opdualag

Intervention Type DRUG

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

Interventions

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Paxalisib

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

Intervention Type DRUG

Opdualag

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

Intervention Type DRUG

Irinotecan (drug)

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

Intervention Type DRUG

Temozolomide (TMZ)

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

Intervention Type DRUG

Eligibility Criteria

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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

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).
Minimum Eligible Age

0 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The Hospital for Sick Children

OTHER

Sponsor Role collaborator

Medical Research Future Fund

OTHER

Sponsor Role collaborator

Kazia Therapeutics Limited

INDUSTRY

Sponsor Role collaborator

C17 Council

OTHER

Sponsor Role collaborator

Bristol-Myers Squibb

INDUSTRY

Sponsor Role collaborator

Stand Up To Cancer

OTHER

Sponsor Role collaborator

Australian & New Zealand Children's Haematology/Oncology Group

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David Ziegler, Prof

Role: STUDY_CHAIR

Sydney Children's Hospital - Australian Study Chair

Daniel Morgenstern, Dr

Role: STUDY_CHAIR

The Hospital for Sick Children - Canadian Study Chair

Locations

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John Hunter Children's Hospital

Newcastle, New South Wales, Australia

Site Status RECRUITING

Sydney Children's Hospital, Randwick

Sydney, New South Wales, Australia

Site Status RECRUITING

The Children's Hospital at Westmead

Sydney, New South Wales, Australia

Site Status RECRUITING

Queensland Children's Hospital

Brisbane, Queensland, Australia

Site Status RECRUITING

Women's and Children's Hospital

Adelaide, South Australia, Australia

Site Status RECRUITING

Royal Hobart Hospital

Hobart, Tasmania, Australia

Site Status RECRUITING

Monash Children's Hospital

Melbourne, Victoria, Australia

Site Status RECRUITING

The Royal Children's Hospital

Melbourne, Victoria, Australia

Site Status RECRUITING

Perth Children's Hospital

Perth, Western Australia, Australia

Site Status RECRUITING

Stollery Children's Hospital

Edmonton, , Canada

Site Status NOT_YET_RECRUITING

CHU Sainte Justine

Montreal, , Canada

Site Status RECRUITING

Children's Hospital of Eastern Ontario

Ottawa, , Canada

Site Status NOT_YET_RECRUITING

The Hospital for Sick Children

Toronto, , Canada

Site Status RECRUITING

BC Children's Hospital

Vancouver, , Canada

Site Status RECRUITING

Countries

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Australia Canada

Central Contacts

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International Study Coordinator

Role: CONTACT

+61 2 9382 1730

International Study Manager

Role: CONTACT

Facility Contacts

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Rebecca Jensen

Role: primary

Sandra Montez

Role: primary

Annie Yeung

Role: primary

Trang Le

Role: primary

Amy Rudge

Role: primary

David Nolan

Role: primary

Amanda St John

Role: primary

Kahlia Fox

Role: primary

Jennifer McConnell

Role: primary

Farah Hassan

Role: primary

Annie Lahaye

Role: primary

Isabelle Laforest

Role: primary

Aiman Siddiqi

Role: primary

Peter Subrt

Role: primary

Other Identifiers

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ANZCHOG2204

Identifier Type: OTHER

Identifier Source: secondary_id

OPTIMISE

Identifier Type: -

Identifier Source: org_study_id

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