Clinical trials

The clinical trials project is central to the translational ambition of PPM.  Clinical trials provide the safety and efficacy data that is critical to the approval of new therapies. For children with rare and untreatable disease, they can provide access to novel treatments where no other options may exist.


In Australia, fewer than 30% of clinical trials are delivered for paediatric patients, significantly impeding the development of interventions targeting conditions affecting children. In putting children at the centre of trial design and development, this project will address that gap, and build capacity to offer novel drugs and gene therapies to those with genetic rare diseases and cancer.  There will also be a focus on adopting innovative trial designs, such as adaptive trials.

The kind of transformative impact clinical trials have on health outcomes for children and young people is exemplified by the drug Nusinersen, which proved so effective in treating the rare genetic condition spinal muscular atrophy (SMA) that the trial was terminated early. Now offered to all SMA patients, the drug has fundamentally changed the way children with SMA are managed, and the expected outcomes for their health.

Associate Director of Research: Operations, Sydney Children's Hospitals Network

Project lead
Ms Cate Smith
Cate Smith

© 2016 Paediatrio