Clinical Trials in Australia

There are several clinical trials for Fabry disease happening in Australia and recruiting Fabry patients. Feel free to discuss participating in any Fabry trial with your own Fabry specialist, and with staff at participating hospitals. If you reside outside the State conducting the trial and are willing to travel, your travel and accommodation costs are covered. To view ALL the various clinical trials for Fabry Disease go to the website

Here are some of the Fabry disease clinical studies recruiting at the moment in Australia.

AVRO-RD-01-201: A Global Clinical TrialOpen-Label, Study of Efficacy and Safety Of AVR-RD-01 for Treatment - Naive Subjects With Classic Fabry Disease Who can participate? Males who have been diagnosed with classic Fabry disease and are not currently receiving enzyme replacement therapy and who have never received chaperone therapy. This is not the full list of eligibility criteria. Find out if you qualify and get more information on this study by contacting your Fabry Clinic. The Perth and Melbourne Fabry Clinics are heading this study in Australia. Further information on the Avrobio clinical study for Fabry Disease Press Here

News from Idorsia Pharmaceuticals based in SwitzerlandA Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects with Fabry Disease The global Phase 3 clinical study ‘MODIFY’ is for patients with Fabry disease. The purpose of the study is to test whether lucerastat monotherapy – the new oral investigational medication – can reduce symptoms of Fabry disease, such as pain and stomach problems and to confirm that it is safe to take.If you are an adult patient with Fabry disease and have neuropathic pain, and you are interested in participating, please contact your doctor or contact a clinical site near you. The Perth and Melbourne Fabry Clinics are heading this study in Australia. Further information about the Idorsia clinical study for Fabry Disease Press Here

Protalix- Pegunigalsidase alfa (PRX-102)Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) Protalix is a once monthly enzyme infusion, designed to provide higher total exposure to alpha-galsctosidase. Further information PRESS HERE

Migalastat – adolescent clinical study Physician Initiated Expanded Access Request for Migalastat in Individual Patients with Fabry Disease Additional clinical studies are currently under consideration, particularly targeting early treatment in young Fabry patients. PRESS HERE

Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD) This study is being conducted via the University of Sydney. Details PRESS HERE

Fabry 400

This is a cardiac MRI study to identify earliest evidence of heart involvement, active in NSW and Victoria

Fabry Registries - there are 3 in total

Fabry Registry - active in all states Fabry Outcome Study (FOS) - active in Victoria Follow Me (for Migalastat and untreated patients) - anticipated to commence in Victoria Q2 2020

There are additional smaller physician-initiated studies which are open in individual centres. Examples are studies of bone health, sleep disturbance and inflammation which are active in Melbourne.


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