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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06208657
Other study ID # OPTIMISE
Secondary ID
Status Not yet recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date March 2024
Est. completion date December 2035

Study information

Verified date January 2024
Source Australian & New Zealand Children's Haematology/Oncology Group
Contact National Study Coordinator
Phone +61293821730
Email SCHN-OPTIMISE@health.nsw.gov.au
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Description:

Both Australia (Zero Childhood Cancer) and Canada (PROFYLE) have developed precision oncology programs for the pediatric population through which samples from childhood/adolescent cancers undergo in depth genetic profiling. OPTIMISE is a companion platform trial, which will link patients to novel targeted agents based on their tumor profile. The trial will have multiple basket arms based on the most common genetically altered pathways the investigators have identified in these childhood cancers. Each arm of the trial will be histopathology agnostic and test a rational, novel combination therapy, to maximise potential clinical benefit.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 82
Est. completion date December 2035
Est. primary completion date December 2030
Accepts healthy volunteers No
Gender All
Age group 0 Years to 21 Years
Eligibility 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 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


Intervention

Drug:
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
Pimasertib
Pimasertib 28mg/m2 oral, twice daily, 28 day cycle, 26 cycles

Locations

Country Name City State
Australia Women's and Children's Hospital Adelaide South Australia
Australia Queensland Children's Hospital Brisbane Queensland
Australia Monash Children's Hospital Melbourne Victoria
Australia Royal Children's Hospital Melbourne Victoria
Australia John Hunter Children's Hospital Newcastle New South Wales
Australia Perth Children's Hospital Perth Western Australia
Australia Sydney Children's Hospital, Randwick Sydney New South Wales
Australia The Children's Hospital at Westmead Sydney New South Wales
Canada CHU Sainte Justine Montréal
Canada The Hospital for Sick Children Toronto
Canada BC Children's Hospital Vancouver

Sponsors (7)

Lead Sponsor Collaborator
Australian & New Zealand Children's Haematology/Oncology Group C17 Council, Children's Cancer Institute Australia (CCIA), Day One Biopharmaceuticals, Inc., Kazia Therapeutics Limited, Medical Research Future Fund, The Hospital for Sick Children

Countries where clinical trial is conducted

Australia,  Canada, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of participants treated with molecularly-targeted agents in each treatment arm. 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. 5 Years
Primary Recommended phase II dose for each treatment arm 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. 3 Years
Primary Objective Response Rate (ORR) for each treatment arm. ORR defined as complete response and partial response, as measured by RECIST, RAPNO, INRC or RECIL in CAYA participants treated with molecularly-targeted agents. 5 Years
Secondary Overall Clinical Benefit Rate (CBR) for each treatment arm 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. 5 Years
Secondary Progression Free Survival (PFS) for each treatment arm. 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. 5 Years
Secondary Incidence of treatment-emergent adverse events for each treatment arm. 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. 5 Years
Secondary Maximum Concentration (Cmax) of molecularly-targeted agents for each treatment arm. Cmax in plasma after the first dose of molecularly-targeted agents in CAYA participants. 5 Years
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