The Perron Institute for Neurological and Translational Science has been approved to participate in two new drug trials for people with Huntington’s disease, 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.