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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.

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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.

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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.

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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

TREATMENT

Phase

PHASE1

PHASE2

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

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

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

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

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/Arm	Intervention/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 Measure	Primary Outcome Description	Primary 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 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
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 Measure	Secondary Outcome Description	Secondary Outcome Time Frame
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
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

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You may be eligible to participate in this trial based on your search.Apply for study

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References

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