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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
Scientific title
Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer
Secondary ID [1] 0 0
OPTIMISE
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Childhood Cancer 0 0
Childhood Solid Tumor 0 0
Childhood Brain Tumor 0 0
Recurrent Cancer 0 0
Refractory Cancer 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants treated with molecularly-targeted agents in each treatment arm.
Timepoint [1] 0 0
5 Years
Primary outcome [2] 0 0
Recommended phase II dose for each treatment arm
Timepoint [2] 0 0
3 Years
Primary outcome [3] 0 0
Objective Response Rate (ORR) for each treatment arm.
Timepoint [3] 0 0
5 Years
Secondary outcome [1] 0 0
Overall Clinical Benefit Rate (CBR) for each treatment arm
Timepoint [1] 0 0
5 Years
Secondary outcome [2] 0 0
Progression Free Survival (PFS) for each treatment arm.
Timepoint [2] 0 0
5 Years
Secondary outcome [3] 0 0
Incidence of treatment-emergent adverse events for each treatment arm.
Timepoint [3] 0 0
5 Years
Secondary outcome [4] 0 0
Maximum Concentration (Cmax) of molecularly-targeted agents for each treatment arm.
Timepoint [4] 0 0
5 Years

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.
Minimum age
0 Years
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/07/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2035
Actual
Sample size
Target
82
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
John Hunter Children's Hospital - Newcastle
Recruitment hospital [2] 0 0
Sydney Children's Hospital, Randwick - Sydney
Recruitment hospital [3] 0 0
The Children's Hospital at Westmead - Sydney
Recruitment hospital [4] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [5] 0 0
Women's and Children's Hospital - Adelaide
Recruitment hospital [6] 0 0
Monash Children's Hospital - Melbourne
Recruitment hospital [7] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [8] 0 0
Perth Children's Hospital - Perth
Recruitment postcode(s) [1] 0 0
- Newcastle
Recruitment postcode(s) [2] 0 0
- Sydney
Recruitment postcode(s) [3] 0 0
- Brisbane
Recruitment postcode(s) [4] 0 0
- Adelaide
Recruitment postcode(s) [5] 0 0
- Melbourne
Recruitment postcode(s) [6] 0 0
- Perth
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Montréal
Country [2] 0 0
Canada
State/province [2] 0 0
Toronto
Country [3] 0 0
Canada
State/province [3] 0 0
Vancouver

Funding & Sponsors
Primary sponsor type
Other
Name
Australian & New Zealand Children's Haematology/Oncology Group
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Children's Cancer Institute Australia (CCIA)
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
The Hospital for Sick Children
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Medical Research Future Fund
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Commercial sector/industry
Name [4] 0 0
Kazia Therapeutics Limited
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Commercial sector/industry
Name [5] 0 0
Day One Biopharmaceuticals, Inc.
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
C17 Council
Address [6] 0 0
Country [6] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
David Ziegler, Prof
Address 0 0
Sydney Children's Hospital - Australian Study Chair
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
National Study Coordinator
Address 0 0
Country 0 0
Phone 0 0
+61293821730
Fax 0 0
Email 0 0
SCHN-OPTIMISE@health.nsw.gov.au
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


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
Name 441 0
David Ziegler
Address 441 0
Kids Cancer Centre Level 1, South Wing, Sydney Children’s Hospital High Street, Randwick NSW 2031
Country 441 0
Australia
Phone 441 0
+612 9382 1730
Fax 441 0
+612 9382 1789
Email 441 0
d.ziegler@unsw.edu.au
Contact person for public queries
Title 442 0
Mx
Name 442 0
National Coordinating Centre
Address 442 0
Kids Cancer Centre Level 1, South Wing, Sydney Children’s Hospital High Street, Randwick NSW 2031
Country 442 0
Australia
Phone 442 0
+61 2 938 21730
Fax 442 0
Email 442 0
SCHN-OPTIMISE@health.nsw.gov.au
Contact person for scientific queries
Title 443 0
Dr
Name 443 0
David Ziegler
Address 443 0
Kids Cancer Centre Level 1, South Wing, Sydney Children’s Hospital High Street, Randwick NSW 2031
Country 443 0
Australia
Phone 443 0
+612 9382 1730
Fax 443 0
+612 9382 1789
Email 443 0
d.ziegler@unsw.edu.au