PROOF-HD Study Enrolling Huntington Disease Patients

Michael Hayden, MBChB, PhD, CEO of Prilenia Therapeutics and a Killam Professor at the University of British Columbia, discusses the PROOF-HD trial.

The PROOF-HD trial is testing the safety and efficacy of pridopidine in patients with early stage Huntington disease.

Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting at around 40 years of age. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death.

As Dr. Hayden explains, the PROOF-HD trial is a pivotal, phase 3 trial being conducted in North America and Europe in collaboration with the Huntington Study Group. The first U.S. subjects were enrolled in October 2020 and enrollment is predicted to end in November 2021. The primary endpoint is Total Functional Capacity (TFC). The TFC scale assesses how people with HD manage their work, finances, daily living, domestic chores, and their care. The treatment period will last up to 78 weeks and there will be an optional open-label extension.

Pridopidine is a selective, high-affinity Sigma-1-receptor (S1R) agonist that has neuroprotective effects in preclinical models of Huntington disease.