Share

Save

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

PHASE1PHASE2RECRUITING

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

info
Simpliy with AI

Study details:

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.

info
Simplify with AI

Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  • 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.
  • 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.
  • Patients must be enrolled on a precision medicine study (i.e. PROFYLE, ZERO or equivalent as agreed with Study Chair.
  • Patients enrolled in a Phase I cohort must have either evaluable or measurable disease.
  • 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.
  • 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.
  • Performance status: Karnofsky performance status (for patients > 16 years of age) or Lansky play score (for patients ≤ 16 years of age) ≥ 50%.
  • Life expectancy ≥ 6 weeks.
  • 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.
  • Adequate organ function.
  • Able to comply with scheduled follow-up and with management of toxicity.
  • Females of childbearing potential must have a negative serum or urine pregnancy test.
  • Fertile males must agree to use adequate contraception during the study and following completion of treatment.
  • Provide a signed and dated informed consent form.
  • Exclusion criteria

  • 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.
  • Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs.
  • Clinically significant, uncontrolled heart disease.
  • Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
  • Presence of any ≥Grade 2 treatment-related toxicity.
  • Major surgery within 21 days of the first dose of investigational drug.
  • Known hypersensitivity to any study drug or component of the formulation.
  • Pregnant or nursing (lactating) females.
  • 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.
  • info
    Simplify with AI

    Eligibility

    Age eligible for study : 0 and older

    Healthy volunteers accepted : No

    Gender eligible for study: All

    Things to know

    Study dates

    Study start: 2024-07-10

    Primary completion: 2030-12-01

    Study completion finish: 2035-12-01

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE1

      PHASE2

    trial

    Trial ID

    NCT06208657

    Intervention or treatment

    DRUG: Paxalisib, Irinotecan, Temozolomide

    DRUG: Pimasertib

    Conditions

    • Childhood Cancer
    • Childhood Solid Tumor
    • Childhood Brain Tumor
    • Recurrent Cancer
    • Refractory Cancer
    Image related to Childhood Cancer
    • Condition: Childhood Cancer, Childhood Solid Tumor and more

    • DRUG: Paxalisib, Irinotecan, Temozolomide and other drugs

    • Sydney, New South Wales, Australia and more

    • Sponsor: Australian & New Zealand Children's Haematology/Oncology Group

    Find a site

    Closest Location:

    The Children's Hospital at Westmead

    Research sites nearby

    Select from list below to view details:

    • The Children's Hospital at Westmead

      Sydney, New South Wales, Australia

    • John Hunter Children's Hospital

      Newcastle, New South Wales, Australia

    • Sydney Children's Hospital, Randwick

      Sydney, New South Wales, Australia

    • Queensland Children's Hospital

      Brisbane, Queensland, Australia

    Loading...

    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/Treatment
    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
    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

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    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
    Recommended phase II dose for each treatment armRecommended 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
    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 outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    Overall Clinical Benefit Rate (CBR) for each treatment armCBR 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
    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
    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
    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

    Frequently Asked Questions

    Please note: some questions and answers are submitted by anonymous patients or using AI, and have not been verified by Clinrol

    No questions submitted. Be the first to ask a question!

    You may be eligible to participate in this trial based on your search.Apply for study
    Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

    References

    Clinical Trials Gov: Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

    Other trails to consider

    Top searched conditions