Targeted therapies for childhood and adolescent cancer

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This trial aims to link pediatric patients with childhood and adolescent cancers to novel targeted therapies based on their tumor genetic profiles. It utilizes precision oncology programs from Australia and Canada, which perform in-depth genetic profiling of cancer samples. The trial features multiple basket arms that focus on common genetically altered pathways in these cancers, allowing for histopathology-agnostic testing of innovative combination therapies to enhance clinical outcomes.

Why it matters

Who can join: the full rules

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 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).

Where it's running

• John Hunter Children's Hospital
  Newcastle, New South Wales, Australia Recruiting
• Sydney Children's Hospital, Randwick
  Sydney, New South Wales, Australia Recruiting
• The Children's Hospital at Westmead
  Sydney, New South Wales, Australia Recruiting
• Queensland Children's Hospital
  Brisbane, Queensland, Australia Recruiting
• Women's and Children's Hospital
  Adelaide, South Australia, Australia Recruiting
• Royal Hobart Hospital
  Hobart, Tasmania, Australia Recruiting

Study contacts

• Study coordinator: International Study Coordinator
• Email: SCHN-OPTIMISE@health.nsw.gov.au
• Phone: +61 2 9382 1730

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.