Trial Review

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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
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

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
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/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/03/2024
Actual
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
A companion platform trial to test novel targeted agents based on the patient's tumor
profile.
Trial website
https://clinicaltrials.gov/ct2/show/NCT06208657
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT06208657