CONNECT-1903: New trial for kids' brain cancer

2021-04-09
A transparent, 3D illustration of a child-sized human head (side-profile) depicts a brain with a mass growing in the rear-right quadrant - the mass has been highlighted bright red to make it stand out; the background is black to increase the contrast.
Aussie kids first in the world to access new international clinical trial for deadly brain tumours.

ANZCHOG is pleased to announce that Australian children will be the first in the world to access a novel drug as a part of a new international clinical trial for a group of deadly brain tumours

Approximately 40-50 Australian children are diagnosed with High Grade Gliomas (HGG) every year.HGG include the universally lethal Diffuse Intrinsic Pontine Glioma (DIPG) and are the leading cause of childhood cancer mortality, as they are poorly responsive to even the most aggressive therapies.

Read more: CONNECT-1903

CONNECT-1903 is the first study to examine if a novel drug larotrectinib can be used in the treatment of children with newly-diagnosed HGG. As larotrectinib targets tumours with a specific cancer-causing mutation (NTRK fusions), only children with this particular genetic change will be eligible for this trial (which can be up to 40% of children diagnosed with HGG in some age groups, such as babies and infants). Australian Principal Investigator, Professor Nick Gottardo explains:

At Perth Children’s Hospital, we are very excited to be the first centre in the world to offer our children access to this promising drug...

Larotrectinib has shown remarkable anti-tumour activity in other cancers with NTRK fusions, so we are delighted to have the opportunity to offer an agent that has the potential to improve outcomes for these children. It is vital that we offer this drug in the regulated setting of a clinical trial, so we can be confident about how well it is tolerated and its effectiveness in treating paediatric HGG.


Dr Raelene Endersby and Professor Nick Gottardo are sitting in a lab surrounded by clinical testing equipment; they smile as they perform their important work.Professor Nick Gottardo with Dr Raelene Endersby, co-heads of the Telethon Kids Institute's Brain Tumour Research Team in Western Australia.

CONNECT-1903 is a clinical trial designed by COllaborative Network for NEuro-oncology Clinical Trials (CONNECT), a recently established United States (U.S)-led consortium who are developing clinical trials for high-risk paediatric brain cancers by combining novel agents with traditional therapies.

Read more: Behind the science | Professor Nick Gottardo

This is the first opportunity for Australian children to participate in a trial run by CONNECT, and paves the way for access to other trials in the expanding CONNECT clinical trial portfolio...

At this stage, three centres will open CONNECT-1903 – Perth Children’s Hospital, Sydney Children’s Hospital and Queensland Children’s Hospital. We are hopeful that we can expand the number of Australian and New Zealand centres that will be able to access CONNECT trials in the future.

-Professor Nick Gottardo


ANZCHOG would like to acknowledge the generous support of CONNECT-1903 co-funders: the Australian Government’s Medical Research Futures Fund, The Kids’ Cancer Project and Love for Lachie.

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