New Local Paediatric Trial Announcement
A Study to Learn More About the Effects and Long-Term Safety of a study drug (an Investigational drug) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old
In exciting news for both the Australian & New Zealand Friedreich ataxia communities, we are now enrolling participants for the BRAVE study, a global Phase 3 study evaluating the effects and safety of a study drug in children with Friedreich ataxia (FA) between the ages of 2 – 15 years. This study is sponsored by Biogen. The design of this global Phase 3 study has been informed by input from previous studies and investigators, health authorities, global medical experts, and the FA community. One of the sites for this important study is at the Murdoch Children’s Research Institute in Melbourne, Australia under the lead of Professor Martin Delatycki.
This is a randomised controlled trial where participants will either receive a daily dose of a study drug or a placebo for one year. The study drug/placebo is a pill (capsule) that is taken by mouth. After this time participants may then have the opportunity to participate in an open label extension study which means all participants will receive study drug. Regular attendance at the Murdoch Children’s Research Institute in Melbourne will be part of the study.
There are several inclusion and exclusion criteria however overall key inclusion criteria are:
Children aged 2- 15 years with a genetically confirmed diagnosis of Friedreich Ataxia and, showing symptoms of Friedreich Ataxia.
There are also criteria for children aged 7 to 15 years regarding their score on a measure of balance and upright stability.
Some of the key exclusion criteria relate to results on various blood and cardiac tests.
If you would like further information on this study we encourage you to visit clinicaltrials.gov Study ID NCT06953583. If you are interested in learning more about this study, please email Varlli Scott at varlli.scott@mcri.edu.au and we will arrange a time to speak to you. If your child wishes to participate in the trial following that discussion, your child will require a screening visit to assess eligibility.
EOI, v1.0_03Jun2025