The Perron Institute for Neurological and Translational Science on the QEII Medical Centre site in Nedlands has been approved to participate in two new drug trials, called PRECISION-HD1 and PRECISION-HD2. Each study tests a separate Huntingtin-lowering (also known as gene silencing) agent. They are run by a company called Wave Life Sciences based in Massachusetts, USA. The studies involve lumbar punctures, MRI scans, assessments and questionnaires.
These are Phase 1b/2a trials, meaning that they are testing an experimental treatment in humans for the first time. The main goal is to assess whether the treatments are safe and well tolerated in the treatment of Huntington’s disease when compared with a placebo control. Participants must be between the ages of 25-65, have early stage Huntington’s disease, and meet additional criteria.
The study has already commenced overseas. Information can be found at https://clinicaltrials.gov/ct2/show/NCT03225833?recrs=a&cond=huntington%27s+disease&draw=2&rank=16
It is expected that together the two studies will involve approximately 96 people worldwide.
If you would like more information, you can call the Perron Institute on 6457 0210 and leave a message for Jo Borrelli, the study coordinator. Please leave a message on the machine if we do not answer when you call. Alternatively you can email Jo at Joanne.Borrelli@perron.uwa.edu.au