A trial with Mivelsiran in Perth

It has been a while since you have heard from us. Fortunately, that's for good reason! We were very busy working on getting DCAA included in a trial and we have great news! Early 2025 you will be able to participate in cAPPricorn-1, a phase II trial by Alnylam with Mivelsiran (formerly known as ALN-APP).

As a DCAA family member myself, I am incredibly excited that for the first time in our lives we are able to participate in a drug trial and we are actually moving forward on a potential treatment for DCAA. The trial does not mean there is a treatment available, since Mivelsiran is a developmental drug and not a treatment, but nonetheless, it is great progress.

In our newest newletter (easier to read on a desktop than mobile phone) you'll find all the information you need about what Mivelsiran does, what a phase II trial looks like, who can participate in the study and why, what participation looks like, genetic testing in the context of a drug trial, family planning and other important things when thinking about participating. Feel free to send the newsletter to family members who might have not received it by email.

There's also other great news in the newsletter, including a number of advocates working together with the research team on a grant that will help improve the work of DCAA family advocates in W.A. That will help us set up a number of things, including another family meeting in early 2025, to discuss the drug trial together and to get your input.

If you have any questions, please contact Samantha Gardener from the research team at s.gardener@ecu.edu.au.