Journal article

Safety, tolerability, and efficacy of PBT2 in Huntington's disease: a phase 2, randomised, double-blind, placebo-controlled trial

D Angus, C Herd, C Stone, J Stout, M Wieler, R Reilmann, CW Ritchie, ER Dorsey, K Helles, E Kayson, D Oakes, HD Rosas, C Vaughan, PK Panegyres, D Ames, A Goh, P Agarwal, A Churchyard, M Murathodizic, P Chua Show all

The Lancet Neurology | ELSEVIER SCIENCE INC | Published : 2015


BACKGROUND: PBT2 is a metal protein-attenuating compound that might reduce metal-induced aggregation of mutant huntingtin and has prolonged survival in a mouse model of Huntington's disease. We aimed to assess the safety, tolerability, and efficacy of PBT2 in patients with Huntington's disease. METHODS: In this 26-week, randomised, double-blind, placebo-controlled trial, adults (≥25 years old) with early-stage to mid-stage Huntington's disease were randomly assigned (1:1:1) by a centralised interactive response system to once daily PBT2 250 mg, PBT2 100 mg, or placebo. Randomisation was stratified by site with a block size of three. Participants, carers, the steering committee, site investig..

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