Waltham, MA– Prilenia Therapeutics B.V. and Ferrer announced the initiation of the confirmatory PRECISE-HD study, a Phase 3 trial evaluating pridopidine in Huntington’s disease.
The study, formally titled Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease, is designed to further assess the efficacy and safety of pridopidine, an investigational oral capsule taken twice daily. Recruitment has begun in the United States, with additional countries expected to follow on a rolling basis later this year.
The randomized, double-blind, placebo-controlled study is expected to enroll 400 participants and generate data to support regulatory evaluation. It will assess the impact of pridopidine on disease progression, functional capacity, motor function, cognition, speech and quality of life.
The PRECISE-HD study will include people living with early- to mid-stage Huntington’s disease, defined as a Total Functional Capacity score of 7 to 13, Total Motor Score of at least 20 and Independence Scale score of 90 percent or lower. The companies said the criteria are intended to include participants who have experienced motor changes or some impact on independent functioning, allowing researchers to evaluate the potential effect of treatment on disease progression compared with placebo.
The study is expected to be conducted at up to 75 sites globally, including in the United States, European Union, United Kingdom and Canada.
Katie Jackson, President/CEO of Help 4 HD International, said, “For families affected by Huntington’s disease, each day is a race against time. There are currently no available treatments to slow the progression of HD, which gradually diminishes every bit of quality of life. Despite these challenges, the therapeutic needs of the HD community remain significantly underserved. Every study aimed at changing this reality is critically important. It is essential that this study be completed promptly so its results—and their potential impact on individuals with HD and their families, who have already waited far too long for progress—can be fully evaluated.”
Victor Sung, M.D., Director of the Division of Movement Disorders at the University of Alabama, Director of the UAB Huntington’s Disease Clinic, Director of the HDSA Center of Excellence and a PRECISE-HD Steering Committee member, said, “Across prior studies, pridopidine has shown meaningful clinical effects in specific circumstancesii, and this study has been carefully designed to confirm those effects, and uniquely incorporates prior learnings and novel elements specifically designed to provide treatment effect clarity.”
PRECISE-HD will include two consecutive stages. The first is a 52-week placebo-controlled stage in which participants will be randomized on a 1:1 basis to receive pridopidine or placebo. That will be followed by a 104-week open-label extension stage, in which all eligible participants will receive pridopidine.
The extension phase will allow researchers to assess any potential effect on disease progression for up to three years in total, compared with matched external control cohorts from longitudinal, multinational observational studies.
The primary endpoint of the study is the change from baseline to Week 52 in the combined Unified Huntington’s Disease Rating Scale score.
