6 results
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…
PRIMARY* To demonstrate the non-inferiority of intravenous-to-oral antibiotic switch therapy in clinically stable neonates with probable bacterial infection compared to a complete course of intravenous antibiotic therapySECONDARY* To describe the…
Primary Objective:To evaluate the effect of sonlicromanol on motor symptom severity in children with genetically confirmed mitochondrial disease affecting oxidative phosphorylation during a 6 month treatment period (GMFM).
The primary objective of this study is to enable continued treatment with the study drug sonlicromanol for patients who have completed study KH176-202 and to investigate the safety and tolerability of sonlicromanol over an 12 months treatment period…
To determine the immune-related progression free survival (irPFS) rate at 21 weeks and objective response rate (irORR) at 30 weeks in patients with pMMR CRC, dMMR mCRC, dMMR EC , dMMR SBC, dMMR SC treated with pembrolizumab combined with ataluren…