The PROOF-HD study, sponsored by Prilenia Therapeutics, is being led by Professor Zosia Miedzybrodzka of the University of Aberdeen. Up to 480 people with early-stage Huntington’s disease will be enrolled across the US, Canada and Europe.
PROOF-HD is a phase 3 clinical trial to investigate the use of pridopidine as a treatment. It requires participants to take a capsule orally twice a day.
The drug, which has been tested already in nearly 1000 HD patients and shown to be safe, is designed to activate a certain receptor which is highly expressed in the brain called the Sigma-1 receptor (S1R). Activation of the S1R triggers mechanisms that are crucial for maintaining neurons function and survival. This may lead to beneficial effects on functional capacity in Huntington’s disease.
Professor Zosia Miedzybrodzka, from the University of Aberdeen, who is leading the study, said: “Huntington’s disease is a serious condition with no known treatments that slow functional decline.
“With no effective treatment currently trials such as PROOF-HD are of huge importance and the promise of effective treatments is really important to families living with the disease.”
Alistair Haw, Chief Executive Officer of Scottish Huntington’s Association, said: “Whilst there are no current treatments that slow the advance of HD, families can be encouraged by the volume of trials currently underway to find the breakthrough we all long for.
“When this breakthrough comes, as we firmly believe it will, it will be thanks to the courage of family members who selflessly volunteer to take part in such trials combined with the expertise of the world’s top researchers, scientists and clinicians.
“We are hugely fortunate so have such a dedicated community in Scotland and we look forward to working with Professor Miedzybrodzka, her team and our HD families as they take this exciting new research project forward.”