PRECISE-HD – a randomized, placebo-controlled, double-blind phase 3 trial in hd evaluating the efficacy and safety of pridopidine from early to mid-stage disease [Conference Abstract]

Sung, Victor, Furr-Stimming, Erin, Rosser, Anne ORCID: https://orcid.org/0000-0002-4716-4753, Kulisevsky, Jaime, Claassen, Daniel O., Hand, Randal, Gianatti, Michele, Chen, Kelly, Gorina, Eduard, Geva, Michal, Hayden, Michael R and Reilmann, Ralf 2026. PRECISE-HD – a randomized, placebo-controlled, double-blind phase 3 trial in hd evaluating the efficacy and safety of pridopidine from early to mid-stage disease [Conference Abstract]. Journal of Neurology, Neurosurgery and Psychiatry 97 (Supp 2) , A97. 10.1136/jnnp-2026-ehdn.220

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Abstract

Background: Pridopidine is an oral, highly selective sigma-1 receptor agonist in development for Huntington’s disease (HD) and amyotrophic lateral sclerosis. PROOF-HD (NCT04556656; EudraCT 2020-002822-10) was a phase 3, randomized, double-blind, placebo-controlled trial assessing pridopidine in early manifest HD. The primary endpoint (change from baseline to week-65 in Total Functional Capacity [TFC]) and key secondary (composite Unified Huntington’s Disease Rating Scale [cUHDRS]) were not met. Among a subgroup of participants off antidopaminergic medications (ADMs), pridopidine showed nominal slowing versus placebo across cUHDRS (LS mean differences +0.41 to +0.46 at Weeks 26–52; all p≤0.035), and Stroop Word Reading (SWR: +3.05 to +3.16; p≤0.042), and exploratory two-year slowing versus propensity-matched ENROLL-HD and TRACK-HD external controls across cUHDRS (59–63%), SWR (81–89%), TFC (62–65%), and Quantitative-Motor (Q-Motor, 78%); all nominal, p<0.005. Aims: PRECISE-HD (NCT07609108) is a confirmatory global, randomized, double-blind, placebo-controlled phase 3 trial evaluating pridopidine (45 mg bid) in people living with HD, from early to mid-stage disease. Methods: A 52-Week double-blind period is followed by a 104-week open-label extension. Eligibility includes adult-onset disease, TFC 7–13, ≥40 CAG repeats, CAG-Age Product (CAP100) ≥95, Total Motor Score (TMS) ≥20, Independence Scale (IS) ≤90%, and no ADM-use for 6-months before screening in ~400 participants randomized 1:1 across up to 75 sites. The primary endpoint is change-from-baseline to Week-52 in cUHDRS. Secondary and exploratory endpoints include function, cognition, motor performance, speech, and plasma biomarkers. Results: Enrollment ongoing. Conclusions: PRECISE-HD evaluates pridopidine in people living with HD from early to mid-stage disease.

Item Type:	Short Communication
Date Type:	Publication
Status:	Published
Schools:	Schools > Medicine
Publisher:	BMJ Publishing Group
ISSN:	0022-3050
Last Modified:	27 Oct 2026 15:03
URI:	https://orca.cardiff.ac.uk/id/eprint/190466

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