RecruitingPHASE1, PHASE2INTERVENTIONAL

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

This study, called OPTIMISE, is for children and teenagers with cancer (up to 21 years old) where standard treatments haven't worked or where there isn't a standard treatment available. It's a special type of trial that uses advanced tests on a patient's cancer cells to understand what makes them grow. Based on these findings, doctors can then choose specific new medications or combinations of medications, like Paxalisib, Opdualag, Irinotecan, and Temozolomide, that are more likely to work for that individual's cancer. The aim is to find more effective and personalised ways to treat childhood cancers, trying out new combinations to give the best possible outcome. This is an early-stage study, meaning these treatments are still being tested.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Australian & New Zealand Children's Haematology/Oncology Group

Enrolment target

Start

10 Jul 2024

Estimated completion

01 Dec 2035

Childhood Cancer Childhood Solid Tumor Childhood Brain Tumor Recurrent Cancer Refractory Cancer

What is this study about?

When children and teenagers get cancer, doctors often try standard treatments that have worked for many others. However, sometimes these treatments don't work, or there isn't a known standard cure. That's where studies like OPTIMISE come in. This study is designed to look very closely at each patient's cancer cells to understand their unique genetic makeup – it's like getting a detailed blueprint of the cancer.

Once doctors have this detailed information, they can then pick specific new medicines or combinations of medicines that are tailored to fight that particular cancer more effectively. This is sometimes called 'precision medicine' because it's about being very precise in how they treat the cancer, rather than a one-size-fits-all approach. By testing these new, targeted treatments, the study hopes to find better ways to help children and teenagers with cancer, especially when other treatments haven't worked.

This study is building on similar work done in Australia and Canada, where advanced genetic testing helps doctors choose treatments. OPTIMISE aims to connect patients with these new, targeted treatments based on what the tests show about their cancer. It's testing several new medicines (like Paxalisib and Opdualag) and how they might work best in combination. The goal is to maximise the chance of a good outcome for each patient.

Key takeaways

This study offers personalised treatments for childhood cancer.
It uses advanced genetic testing of your cancer to guide treatment choices.
It's for children and teenagers whose cancer hasn't responded to standard care.
New drug combinations are being tested (Phase 1 & 2).
You'll be closely monitored for any side effects and treatment response.

Who may be eligible?

This study is for children and teenagers under 21 who have cancer that hasn't responded to standard treatments, or for which there isn't a widely accepted treatment. In special cases, adults over 21 with a type of cancer usually found in children might be able to join, but this needs approval from the study doctor.

Before taking part, you would need to have had specific genetic tests done on your cancer through another special programme (like PROFYLE or ZERO). You'll also need to be well enough to take part, meaning you've recovered from previous cancer treatments and have good general health, including your organ functions. There are also specific checks to make sure your cancer can be measured during the study.

There are also some reasons why someone might not be able to join. For example, if your brain or spinal cord tumour is getting worse quickly and needs urgent treatment, or if you're already taking high doses of certain medications that could interfere with the study. The study team will review all your medical information to see if this study is right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you under 21 with cancer that hasn't responded to standard treatments?
Have you had special genetic tests on your cancer through a specific programme?
Are you generally well enough, and have you recovered from previous cancer treatments?
Are you able to attend follow-up appointments?
Is your medical team supportive of you considering this type of study?

Answer every question to see your result.

What does participation involve?

If you join this study, you won't need extra tests beyond what is usually done for your cancer. Your regular hospital will do these tests, and the results will be sent to the study team. You'll continue to have regular check-ups to see if the treatment is working and to manage any side effects. The exact number of visits will depend on the treatment you receive. You'll need to be able to make it to these scheduled follow-ups. The treatments being tested are designed to target specific features of your cancer, chosen after genetic tests. The total length of your participation will depend on how you respond to the treatment and what the study doctors advise.

Potential risks and benefits

Taking part in a study like this might offer a chance to try new treatments that aren't yet widely available, which could potentially help if standard treatments haven't worked. However, these are new treatments, so there's always a possibility of unknown side effects, or that the treatment might not work for everyone. The study team will carefully monitor you for any side effects and manage them as best they can. You are always free to ask questions and discuss any concerns. Importantly, you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (14)

John Hunter Children's Hospital
Verified postcode
Newcastle, Australia· Recruiting
Sydney Children's Hospital, Randwick
Verified postcode
Sydney, Australia· Recruiting
The Children's Hospital at Westmead
Verified postcode
Sydney, Australia· Recruiting
Queensland Children's Hospital
Verified postcode
Brisbane, Australia· Recruiting
Women's and Children's Hospital
Verified postcode
Adelaide, Australia· Recruiting
Royal Hobart Hospital
Verified postcode
Hobart, Australia· Recruiting
Monash Children's Hospital
Verified postcode
Melbourne, Australia· Recruiting
The Royal Children's Hospital
Verified postcode
Melbourne, Australia· Recruiting
Perth Children's Hospital
Verified postcode
Perth, Australia· Recruiting
Stollery Children's Hospital
Verified postcode
Edmonton, Canada· Not yet recruiting
CHU Sainte Justine
Verified postcode
Montreal, Canada· Recruiting
Children's Hospital of Eastern Ontario
Verified postcode
Ottawa, Canada· Not yet recruiting

Common questions

What kind of cancer is this study for?

It's for children and teenagers (up to 21) with various types of cancer that haven't responded to usual treatments.

How do they choose which medicine I get?

Doctors use special tests on your cancer cells to create a 'profile,' then pick the medicine most likely to work for your specific cancer.

Are these treatments new?

Yes, they are new and being tested in this early-stage study to see how safe and effective they are.

Do I have to travel for this study?

Many standard tests can be done at your local hospital, with results sent to the study team.

Can I stop participating in the study?

Yes, you can choose to leave the study at any time, and it won't affect your medical care.

How to find out more

International Study Coordinator

schn-optimise@health.nsw.gov.au +61 2 9382 1730 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.