3 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…
The primary objective is to test the hypothesis that rhPTH(1-84) treatment can result in superior improvements in the symptoms of hypoparathyroidism as assessed by the Hypoparathyroidism Symptom Diary (HPT-SD) symptom subscale compared with standard…
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…