22 August 2019

Recruiting for Huntington’s clinical trials in WA

The Perron Institute at the QEII Medical Centre in Nedlands is recruiting participants for two Huntington’s Disease clinical trials.

The studies are called PRECISION-HD1 and PRECISION-HD2 and are Phase 1b/2a trials. The Institute is one of several centres in Australia involved in these international studies run by a company called Wave Life Sciences based in Massachusetts, USA.

Perron Institute consultant neurologist Dr Carolyn Orr is leading the WA element of the research. She said the aim was to confirm whether an experimental drug designed by scientists in the USA could safely alter the path of the disease by silencing the mutant version of the Huntington’s gene.

Huntington’s Disease, caused by an inherited defect in a single gene, results in severe impacts on functional abilities such as control of movement as well as cognitive and psychiatric impairment. It is autosomal dominant which means that if a parent has the gene, each son or daughter has a fifty-fifty (one in two) chance of inheriting it.

Most people with Huntington’s develop signs and symptoms in their 40s or 50s but the disease may emerge earlier or later in life.

“The gene was pinpointed some 20 years ago. While there are therapies to help manage symptoms, there is yet no available treatment to halt or reverse the course of the disease.

“Now, with advances in molecular genetics, we are hopeful that a therapy is on the horizon. The current study will assess whether the experimental therapy is safe and well tolerated.

“Screening to find suitable candidates for the proposed clinical trial is the first stage of the recruitment process and is underway,” Dr Orr said.

Participants in this randomised and placebo-controlled trial must be between the ages of 25-65, have early stage Huntington’s disease and meet additional criteria.

For further information: contact the Perron Institute on 08 6457 0210 and leave a message for Study Coordinator Jo Borrelli or email: Joanne.Borrelli@perron.uwa.edu.au.