RecruitingPhase 1/2

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

Australia90 participantsStarted 2024-07-10

Plain-language summary

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

Who can participate

Age range0 Years – 21 Years

SexALL

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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.

Exclusion criteria

✕. 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.
✕. 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.
✕. 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.
✕. Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
✕. 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.
✕. 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 drug(s).

What they're measuring

Number of participants treated with molecularly-targeted agents in each treatment arm.

Timeframe: 5 Years

Recommended phase II dose for each treatment arm

Timeframe: 3 Years

Objective Response Rate (ORR) for each treatment arm.

Timeframe: 5 Years

Trial details

NCT IDNCT06208657

SponsorAustralian & New Zealand Children's Haematology/Oncology Group

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2030-12

Contact for this trial

International Study Coordinator

+61 2 9382 1730 SCHN-OPTIMISE@health.nsw.gov.au

View on ClinicalTrials.gov More Childhood Cancer trials