5 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…
This study is intended to establish a dose response rate of MR30507/09 with respect to respiratory effects compared to MR30365/07, fentanyl and placebo..
The aim of the main study is to compare the effects of MR30365/07 and fentanyl on ventilation and analgesic responses to noxious electrical and heat stimulation in healthy volunteers, by performing pharmacokinetic / pharmacodynamics (PK/PD) modeling…
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…