Professor Carolyn Sue
Kolling Institute, University of Sydney
Creating an Australian Clinical Trials Network for Mitochondrial Disease (mito)
This project will support the activities of the Australian Clinical Trials Network for Mitochondrial Disease (ACTN-MD) and the running of a clinical trial assessing the effectiveness of idebenone on improving vision in patients with mitochondrial disease.
The first clinical trial to provide a solid evidence for disease modification in mito was a randomised placebo controlled trial of idebenone in Leber’s Hereditary Optic Neuropathy (LHON). It was found that patients with visual acuities benefited from idebenone treatment.
Leber hereditary optic neuropathy (LHON) is a maternally-inherited progressive disease typically affecting one eye, then the other eye within a six to twelve-month period, eventually resulting in irreversible loss of visual acuity and blindness.
By setting up the ACTN-MD, Professor Sue and her team have created a sustainable framework and will conduct efficient studies to provide access to patients with mito to clinical trials.
This project will improve the lives of many Australians who suffer from these disorders and will also allow training of junior staff in clinical research practice.