3 results
The study aims to limit the use of anthracyclines and to reduce the dose of ATRA. Another aim is to stratify treatment by risk group: standard risk - WBC <10 x 109/l : high risk - WBC >=10 x 109/l. Furthermore this study aims to…
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: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…