Project summary
The Clinical Trials project will underpin the translational ambition of Luminesce Alliance by building capacity to offer novel drugs and gene therapies to children with genetic rare diseases and cancer, utilising innovative trial design including adaptive trials.
Clinical trials are fundamental to the development of medical innovations, yet only around one third of clinical trials involve children, greatly impeding the development of interventions for paediatric conditions. In putting children at the centre of trial design and development, this project will address that gap.
What is the issue for NSW?
Clinical trials are a fundamental step in the process of translating medical innovations from the research proof of concept stage to use in clinical settings. Clinical trials provide the safety and efficacy data that is critical to the approval of new therapies; for patients with rare and untreatable disease, they also 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 that would benefit children, particularly those suffering from genetic rare diseases and cancers that only affect children.
Providing access to novel therapies for children with rare genetic disease and cancer has the capacity to transform their health outcomes significantly. While trial cohorts may be limited to 2-3 children at one time, the outcome of a successful clinical trial for a novel therapy has the potential to rapidly and profoundly impact the affected population, health service delivery, and the community more broadly.
What does the research aim to do and how?
This project will leverage recent investments by the NSW government in world class infrastructure for the delivery of paediatric trials, providing additional resources to deliver novel drug and gene therapy clinical trials to children with genetic rare disease and cancer. The project will support personnel to lead the development and delivery of innovative trial methodologies and protocol design suitable to the small cohorts (including n=1) that apply to many paediatric rare diseases; and establish an education, implementation and service model to deliver 2-3 early phase trials per year for the benefit of children with genetic rare disease and cancer.
Read more about the work of Luminesce Alliance.
Partnering institutions
- Children’s Cancer Institute
- University of NSW (UNSW)
- Children’s Medical Research Institute
- Sydney Children’s Hospitals Network
- University of Sydney