5 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 objective: To demonstrate that QVA149 (110/50 *g o.d.) is at least non-inferior to salmeterol/fluticasone (50/500 *g b.i.d.) in terms of rate of COPD exacerbations.Secondary objectives: Superiority in terms of exacerbation rate. Time to…
The objectives of this are to establish the safety, tolerability, and efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in HV and TED patients over a dose range of 3.0 to 20.0 mg/kg.
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 objectives:Period 1:The primary objective is to assess the long-term (up to 2 years) safety and tolerability of two doses of PXT3003.Period 2:For patients continuing after V9, the main objective will be to offer patients the opportunity…