Optimise viewing for

Toggle between patient and health professional views. The patient version is simplified to help people easily find suitable trials, while the professional view provides full detail.

RecruitingLast updated: 26 February 2026

OPTIMISE: This clinical trial will link children and young adults to new targeted therapies based on their cancer typeOptimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

Trial purpose

Medical clipboardCancer treatment

Tumor type

Multi-Cancer Multi-Cancer

Age

People0 - 21

Trial acronym

OPTIMISE

Clinical summary

Summary

Both Australia (Zero Childhood Cancer) and Canada (PROFYLE) have developed precision oncology programs for the paediatric population through which samples from childhood and adolescent cancers undergo in-depth genetic profiling.

The OPTIMISE study is a companion platform trial, which will link patients to novel (new) targeted agents (treatments) based on their tumour 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, new combination therapy, to maximise potential clinical benefit. 

Conditions

This trial is treating people with childhood and adolescent cancers, including solid cancers, central nervous system tumours and lymphomas that have progressed despite prior standard treatment or for which no effective standard treatment exists

Eligibility

Inclusion

  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

  1. Patients with symptomatic central nervous system (CNS) primary or metastatic tumours who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease. Patients on stable doses of corticosteroids for at least 7 days prior to receiving study drug may be included.
  2. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhoea, or malabsorption syndrome) - only for arms that include orally administered therapeutic agents.
  3. Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias, e.g., ventricular, supraventricular, nodal arrhythmias, or conduction abnormality), unstable ischemia, congestive heart failure within 12 months of screening.
  4. Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
  5. Major surgery within 21 days of the first dose of investigational drug. Gastrostomy, ventriculo-peritoneal shunt, endoscopic ventriculostomy, tumour biopsy and insertion of central venous access devices are not considered major surgery, but for these procedures, a 48-hour interval must be maintained before the first dose of the investigational drug is administered.
  6. Known hypersensitivity to any study drug or component of the formulation.
  7. Pregnant or nursing (lactating) females.
  8. 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 drug(s).

Inclusion

  • You have previously participated in a certain specific clinical trial.
  • You have had treatment, but your cancer has come back (relapsed or recurrent).
  • You have had treatment but your cancer has gotten worse or has not responded to the treatment you have been given.

Exclusion

  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

Recruiting hospitals

InformationTell us if you find this trial availability is not accurate.Report inaccuracy

More information

Trial Identifiers

Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:

Trial sponsor

Australian & New Zealand Children's Haematology/Oncology Group

Scientific Title

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

Get Support

Peer Connect

You might find it helpful to speak to someone who has 'been there before'. Our Peer Connect program can provide one-on-one phone support from someone who understands what you're going through and has clinical trials experience.

Know more about Peer Connect

Contact cancer support

If you need cancer information and practical support for yourself, a carer, family or friend, contact Cancer Council to speak with an experienced health professional on 13 11 20.

Get support

Multilingual information

Learn more about clinical trials through this collection of resources in languages other than English.

View information