4 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…
Primary Objective: To study which test or which combination of tests has the best predictive value at baseline level for a positive effect of the ITB test-infusion, rated by the patient on the Patient-Global Impression of Change (P-GIC). Secondary…
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)
Phase I study to investigate safety of the combination indomethacin and two platinum-based chemotherapy regimens in patients with advanced cancers.