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Recruiting Phase 1/2

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

NCT06208657 · tracked via the Priya Life Science Canada tracker
Sponsor
Australian & New Zealand Children's Haematology/Oncology Group
Phase
Phase 1/2
Started
2024-07-10
Last updated
2026-01-28

Condition(s) studied

Childhood CancerChildhood Solid TumorChildhood Brain TumorRecurrent CancerRefractory Cancer

Investigational drug(s) / intervention(s)

Paxalisib →Opdualag →Irinotecan (drug) →Temozolomide (TMZ) →

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

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

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

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

Study summary

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

Eligibility

Sex
ALL
Min age
0 Years
Max age
21 Years
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: 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).

Primary outcome measure(s)

  • Number of participants treated with molecularly-targeted agents in each treatment arm. — 5 Years
    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.
  • Recommended phase II dose for each treatment arm — 3 Years
    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.
  • Objective Response Rate (ORR) for each treatment arm. — 5 Years
    ORR defined as complete response and partial response, as measured by RECIST, RAPNO, INRC or RECIL in CAYA participants treated with molecularly-targeted agents.

Trial sites (14)

FacilityCityRegionStatus
John Hunter Children's Hospital Newcastle New South Wales Recruiting
Sydney Children's Hospital, Randwick Sydney New South Wales Recruiting
The Children's Hospital at Westmead Sydney New South Wales Recruiting
Queensland Children's Hospital Brisbane Queensland Recruiting
Women's and Children's Hospital Adelaide South Australia Recruiting
Royal Hobart Hospital Hobart Tasmania Recruiting
Monash Children's Hospital Melbourne Victoria Recruiting
The Royal Children's Hospital Melbourne Victoria Recruiting
Perth Children's Hospital Perth Western Australia Recruiting
Stollery Children's Hospital Edmonton Canada Not Yet Recruiting
CHU Sainte Justine Montreal Canada Recruiting
Children's Hospital of Eastern Ontario Ottawa Canada Not Yet Recruiting
The Hospital for Sick Children Toronto Canada Recruiting
BC Children's Hospital Vancouver Canada Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06208657 on ClinicalTrials.gov ↗ ← All trials in Canada