4 results
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…
The primary objective is to evaluate the inhalation process and the overall satisfaction with the use of the Investigational Medicinal Products (IMPs). The secondary objectives are to determine the subjective effects after pulmonary administration…
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…
To assess the feasibility of using a subtherapeutic dose of piracetam as a marker of adherence to therapy by determining whether this subtherapeutic dose produces a detectable concentration in the urine