5 results
Main Objective:Assess the efficacy of eculizumab as compared with placebo in the treatment of refractory gMG based on the improvement in the MG specific Activities of Daily Living profile (MG-ADL).Secondary Objectives: - Safety and tolerability of…
Primary Objective: To investigate the effect of supplementation of a single high dose of vitamin D (400.000 IU) on 1. the percentage of neutrophils in induced sputum, in non-atopic asthmatic patients with neutrophilic airway inflammation (…
Study AG348-C-018 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β-TDT. The primary objective of the study is to compare the…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused
Study AG348-C-017 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β NTDT. The primary objective of the study is to compare the…