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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06208657
Additional trial details provided through ANZCTR are available at the end of this record.
Registration number
NCT06208657
Ethics application status
Date submitted
12/12/2023
Date registered
17/01/2024
Titles & IDs
Public title
Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer
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Scientific title
Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer
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Secondary ID [1]
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OPTIMISE
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Childhood Cancer
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Childhood Solid Tumor
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Childhood Brain Tumor
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Recurrent Cancer
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Refractory Cancer
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Condition category
Condition code
Other
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0
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Paxalisib, Irinotecan, Temozolomide
Treatment: Drugs - Pimasertib
Experimental: Arm A Paxalisib - Drug: Irinotecan Drug: Temozolomide Drug: Paxalisib Irinotecan 50mg/m2/day, intravenous, on days 1-5, 28 day cycle, 13 cycles Temozolomide 150mg/m2/day, oral, on days 1-5, 28 day cycle, 13 cycles Paxalisib 21mg/m2 oral, daily, 28 day cycle, 13 cycles
Treatment: Drugs: Paxalisib, Irinotecan, Temozolomide
Irinotecan 50mg/m2/day, intravenous, on days 1-5, 28 day cycle, 13 cycles Temozolomide 150mg/m2/day, oral, on days 1-5, 28 day cycle, 13 cycles Paxalisib 21mg/m2 oral, daily, 28 day cycle, 13 cycles
Treatment: Drugs: Pimasertib
Pimasertib 28mg/m2 oral, twice daily, 28 day cycle, 26 cycles
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of participants treated with molecularly-targeted agents in each treatment arm.
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Assessment method [1]
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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.
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Timepoint [1]
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5 Years
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Primary outcome [2]
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Recommended phase II dose for each treatment arm
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Assessment method [2]
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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.
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Timepoint [2]
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3 Years
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Primary outcome [3]
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Objective Response Rate (ORR) for each treatment arm.
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Assessment method [3]
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ORR defined as complete response and partial response, as measured by RECIST, RAPNO, INRC or RECIL in CAYA participants treated with molecularly-targeted agents.
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Timepoint [3]
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5 Years
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Secondary outcome [1]
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Overall Clinical Benefit Rate (CBR) for each treatment arm
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Assessment method [1]
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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.
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Timepoint [1]
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5 Years
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Secondary outcome [2]
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Progression Free Survival (PFS) for each treatment arm.
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Assessment method [2]
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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.
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Timepoint [2]
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5 Years
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Secondary outcome [3]
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Incidence of treatment-emergent adverse events for each treatment arm.
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Assessment method [3]
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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.
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Timepoint [3]
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5 Years
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Secondary outcome [4]
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Maximum Concentration (Cmax) of molecularly-targeted agents for each treatment arm.
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Assessment method [4]
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Cmax in plasma after the first dose of molecularly-targeted agents in CAYA participants.
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Timepoint [4]
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5 Years
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Eligibility
Key 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.
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Minimum age
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Years
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Maximum age
21
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Patients with symptomatic CNS primary or metastatic tumors who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease.
2. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs.
3. Clinically significant, uncontrolled heart disease.
4. Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
5. Presence of any =Grade 2 treatment-related toxicity.
6. Major surgery within 21 days of the first dose of investigational drug.
7. Known hypersensitivity to any study drug or component of the formulation.
8. Pregnant or nursing (lactating) females.
9. 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 drugs.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
10/07/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2035
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Actual
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Sample size
Target
82
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
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Recruitment hospital [1]
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John Hunter Children's Hospital - Newcastle
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Recruitment hospital [2]
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Sydney Children's Hospital, Randwick - Sydney
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Recruitment hospital [3]
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The Children's Hospital at Westmead - Sydney
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Recruitment hospital [4]
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Queensland Children's Hospital - Brisbane
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Recruitment hospital [5]
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Women's and Children's Hospital - Adelaide
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Recruitment hospital [6]
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Monash Children's Hospital - Melbourne
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Recruitment hospital [7]
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Royal Children's Hospital - Melbourne
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Recruitment hospital [8]
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Perth Children's Hospital - Perth
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Recruitment postcode(s) [1]
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- Newcastle
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Recruitment postcode(s) [2]
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- Sydney
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Recruitment postcode(s) [3]
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- Brisbane
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Recruitment postcode(s) [4]
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0
- Adelaide
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Recruitment postcode(s) [5]
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- Melbourne
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Recruitment postcode(s) [6]
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- Perth
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Recruitment outside Australia
Country [1]
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Canada
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State/province [1]
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Montréal
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Country [2]
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Canada
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State/province [2]
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Toronto
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Country [3]
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Canada
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State/province [3]
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Vancouver
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Funding & Sponsors
Primary sponsor type
Other
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Name
Australian & New Zealand Children's Haematology/Oncology Group
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Address
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Country
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Other collaborator category [1]
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0
Other
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Name [1]
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Children's Cancer Institute Australia (CCIA)
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Address [1]
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0
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Country [1]
0
0
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Other collaborator category [2]
0
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Other
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Name [2]
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The Hospital for Sick Children
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Address [2]
0
0
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Country [2]
0
0
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Other collaborator category [3]
0
0
Other
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Name [3]
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0
Medical Research Future Fund
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Address [3]
0
0
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Country [3]
0
0
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Other collaborator category [4]
0
0
Commercial sector/industry
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Name [4]
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Kazia Therapeutics Limited
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Address [4]
0
0
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Country [4]
0
0
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Other collaborator category [5]
0
0
Commercial sector/industry
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Name [5]
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Day One Biopharmaceuticals, Inc.
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Address [5]
0
0
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Country [5]
0
0
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Other collaborator category [6]
0
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Other
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Name [6]
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C17 Council
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Address [6]
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0
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Country [6]
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0
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Ethics approval
Ethics application status
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Summary
Brief summary
A companion platform trial to test novel targeted agents based on the patient's tumor profile.
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Trial website
https://clinicaltrials.gov/study/NCT06208657
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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David Ziegler, Prof
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Address
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Sydney Children's Hospital - Australian Study Chair
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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National Study Coordinator
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Address
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Country
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Phone
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+61293821730
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Fax
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Email
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SCHN-OPTIMISE@health.nsw.gov.au
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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.
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06208657
Additional trial details provided through ANZCTR
Accrual to date
Recruiting in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC
Recruitment hospital [1]
180
Queensland Children's Hospital
Recruitment hospital [2]
181
Sydney Children's Hospital
Recruitment hospital [3]
182
The Children's Hospital at Westmead
Recruitment hospital [4]
183
John Hunter Children's Hospital
Recruitment hospital [5]
184
Monash Children’s Hospital
Recruitment hospital [6]
185
The Royal Childrens Hospital
Recruitment hospital [7]
186
Perth Children's Hospital
Recruitment hospital [8]
187
Womens and Childrens Hospital
Recruitment postcode(s) [1]
179
4101
Recruitment postcode(s) [2]
180
2031
Recruitment postcode(s) [3]
181
2145
Recruitment postcode(s) [4]
182
2305
Recruitment postcode(s) [5]
183
3168
Recruitment postcode(s) [6]
184
3052
Recruitment postcode(s) [7]
185
6009
Recruitment postcode(s) [8]
186
5006
Funding & Sponsors
Primary sponsor
Other Collaborative groups
Primary sponsor name
Australia and New Zealand Children's Haematology and Oncology Group (ANZCHOG)
Primary sponsor address
27 - 31 Wright St, Clayton VIC 3168
Primary sponsor country
Australia
Other collaborator category [1]
98
Hospital
Name [1]
98
The Hospital for Sick Children
Address [1]
98
170 Elizabeth St, Toronto, ON M5G 1E8, Canada
Country [1]
98
Canada
Other collaborator category [2]
99
Other
Name [2]
99
Children's Cancer Institute Australia (CCIA)
Address [2]
99
Lowy Cancer Research Centre, C25/9 High St, Kensington NSW 2033
Country [2]
99
Australia
Other collaborator category [3]
100
Government body
Name [3]
100
Australian Department of Health and Aged Care, Medical Research Future Fund (MRFF)
Address [3]
100
Department of Health GPO Box 9848 Canberra ACT 2601 Australia
Country [3]
100
Australia
Other collaborator category [4]
101
Commercial sector/Industry
Name [4]
101
Kazia Therapeutics Limited
Address [4]
101
Three International Towers Level 24, 300 Barangaroo Avenue Sydney NSW 2000, Australia
Country [4]
101
Australia
Other collaborator category [5]
102
Commercial sector/Industry
Name [5]
102
Day One Biopharmaceuticals, Inc.
Address [5]
102
2000 Sierra Point Parkway, Suite 501 Brisbane, CA 94005
Country [5]
102
United States of America
Other collaborator category [6]
103
Other
Name [6]
103
C17 Council
Address [6]
103
Edmonton Clinic Health Academy, 11405 87 Ave NW #3-590, Edmonton, AB T6G 1C9, Canada
Country [6]
103
Canada
Ethics approval
Ethics application status
Approved
Ethics committee name [1]
76
Sydney Children's Hospitals Network Human Research Ethics Committee
Address [1]
76
The Children’s Hospital at Westmead, Kids Research Building, Corner Hawkesbury Road and Hainsworth Street, Westmead NSW 2145
Country [1]
76
Australia
Date submitted for ethics approval [1]
76
26/06/2023
Approval date [1]
76
13/07/2023
Ethics approval number [1]
76
2023/ETH00222
Public notes
Contacts
Principal investigator
Title
441
0
Dr
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Name
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David Ziegler
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Address
441
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Kids Cancer Centre Level 1, South Wing, Sydney Children’s Hospital High Street, Randwick NSW 2031
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Country
441
0
Australia
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Phone
441
0
+612 9382 1730
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Fax
441
0
+612 9382 1789
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Email
441
0
d.ziegler@unsw.edu.au
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Contact person for public queries
Title
442
0
Mx
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Name
442
0
National Coordinating Centre
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Address
442
0
Kids Cancer Centre Level 1, South Wing, Sydney Children’s Hospital High Street, Randwick NSW 2031
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Country
442
0
Australia
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Phone
442
0
+61 2 938 21730
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Fax
442
0
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Email
442
0
SCHN-OPTIMISE@health.nsw.gov.au
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Contact person for scientific queries
Title
443
0
Dr
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Name
443
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David Ziegler
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Address
443
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Kids Cancer Centre Level 1, South Wing, Sydney Children’s Hospital High Street, Randwick NSW 2031
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Country
443
0
Australia
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Phone
443
0
+612 9382 1730
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Fax
443
0
+612 9382 1789
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Email
443
0
d.ziegler@unsw.edu.au
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