2 results
Approved WMOCompleted
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…
Approved WMOCompleted
Cohort 1 (HER2-positive/amplified MBC): MCLA-128 + trastuzumab ± vinorelbine Primary objective:• Evaluate efficacy of MCLA-128 combined with trastuzumab ± vinorelbine in terms of clinical benefit rate (CBR) at 24 weeks based on RECIST 1.1 (per…