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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT06208657
Registration number
NCT06208657
Ethics application status
Date submitted
12/12/2023
Date registered
17/01/2024
Date last updated
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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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
Experimental: Arm B Pimasertib - Drug: Pimasertib Pimasertib 28mg/m2 oral, twice daily, 28 day cycle, 26 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/Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Not yet 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
1/03/2024
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Actual
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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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- 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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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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Country [1]
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Other collaborator category [2]
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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]
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Country [2]
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Other collaborator category [3]
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Other
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Name [3]
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Medical Research Future Fund
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Address [3]
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Country [3]
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Other collaborator category [4]
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Commercial sector/Industry
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Name [4]
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Kazia Therapeutics Limited
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Address [4]
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Country [4]
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Other collaborator category [5]
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Commercial sector/Industry
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Name [5]
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Day One Biopharmaceuticals, Inc.
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Address [5]
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Country [5]
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Other collaborator category [6]
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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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Country [6]
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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/ct2/show/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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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT06208657
Download to PDF