4 results
Primary objective:To assess the efficacy of PXT3003 compared to Placebo on the disability measured by the ONLS score in CMT1A patients treated for 15 months.Secondary objectives:- To assess the efficacy of PXT3003 compared to Placebo on clinical and…
Primary ObjectivePart1: To evaluate the safety and tolerability of intravenous (IV) administration of AL001 over up to 96 weeks in asymptomatic and symptomatic carriersof a granulin (GRN) mutation causative of frontotemporal dementia (FTD) and in…
The primary objective of this study is: Part 1: To evaluate the efficacy of AL001 compared with placebo as measured by CDR® plus NACC FTLD-SB.Part 2: To assess the long-term safety and tolerability of AL001 in participants who have completed 96…
Primary:To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol) compared to placebo in subjects with CMT1A.Secondary: To evaluate the safety and tolerability of PXT3003…