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)
To evaluate the diagnostic performance and safety of metal allergens proposed for inclusion in Metal Panel T.R.U.E. Test. The study will compare the diagnostic performance (primary) and safety (secondary) of ascending patch test doses of aluminum,…
To demonstrate a 25% increase of docetaxel in tumor tissue after intravenous with CriPec® docetaxel compared to Taxotere®. Additionally, systemic PK profile and adverse events will also be evaluated.
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…