5 results
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…
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 Objective:To assess the effect of MEDI0382 on hepatic glycogen levels versus placebo after 28 days (Part A) and 35 days (Part B) of treatment* Secundary Objective: - To assess the effect of MEDI0382 on hepatic glycogen levels versus…
The objective of this study is to assess the safety and effectiveness of the WISE System (providing LV pacing) when used in conjunction with a co-implanted system (ICD, pacemaker, CRT-P, or CRT-D) for bi-ventricular (BiV) pacing for CRT in heart…
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…