Pridopidine in early-stage manifest Huntington’s disease: a phase 3 trial
Ralf Reilmann; Andrew Feigin; Anne Rosser; Sandra K. Kostyk; Carsten Saft; Yaël C. Cohen; Henk Schuring; Randal Hand · 2025 · Nature Medicine
WASTE classifies this as Replication Failure · AI classification, approximate
A previously reported effect did not replicate here — verify it holds before you build on it.
Abstract
Huntington's disease (HD) is a rare, neurodegenerative disorder for which only symptomatic treatments are available. The PROOF-HD study was a randomized, double-blind, placebo-controlled phase 3 trial evaluating the efficacy and safety of pridopidine, a selective Sigma-1 receptor agonist, in HD. The primary and key secondary endpoints, change in total functional capacity (TFC) and composite Unified Huntington's Disease Rating Scale (cUHDRS) score at week 65, were not met in the overall population. The TFC least-squares mean difference between pridopidine and placebo was -0.18 (95% confidence i
Abstract by Ralf Reilmann; Andrew Feigin; Anne Rosser; Sandra K. Kostyk; Carsten Saft; Yaël C. Cohen; Henk Schuring; Randal Hand, Nature Medicine (2025) — licensed CC BY 4.0.
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Metadata source: OpenAlex · DOI 10.1038/s41591-025-03920-3