PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD

Summary

This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).

Timeline

Start

2020-10-16

Primary completion

2023-03-14

Completion

2024-03-21

Outcome

Missed primary endpoint

registry analysis (superiority test); Pridopidine vs Placebo; p = 0.1670; Mean Difference (Final Values) -0.22 (95% CI -0.54 to 0.09) NCT04556656 ↗

registry analysis (superiority test); Pridopidine vs Placebo; p = 0.1598; Mean Difference (Final Values) -0.23 (95% CI -0.55 to 0.09) NCT04556656 ↗

paper 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. PMID 40913168 ↗

release “The PROOF-HD study did not meet its overall primary or secondary endpoints in the full population” prilenia.com ↗

Publications

• Reilmann R, Feigin A, Rosser AE, Kostyk SK, Saft C, Cohen Y, Schuring H, Hand R, Tan AM, Chen K, Feng W, Navon-Perry L, Cruz-Herranz A, Syltevik C, Boot D, Squitieri F, Kayson E, Mehra M, Goldberg YP, Geva M, Hayden MR; PROOF-HD study investigators. Pridopidine in early-stage manifest Huntington's disease: a phase 3 trial. Nat Med. 2025 Nov;31(11):3780-3789. doi: 10.1038/s41591-025-03920-3. Epub 2025 Sep 5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Pridopidine	Small molecule	45 mg	—

Indications