5 results
To evaluate the efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Core StudyPrimary objective• To compare the efficacy of E5501 (in addition to standard of care) to eltrombopag (in addition to standard of care) for the treatment of adult subjects with chronic immune thrombocytopenia (idiopathic thrombocytopenic…
Core Study:Primary objective: • To demonstrate that the efficacy of E5501 (in addition to standard of care) is superior to placebo (in addition to standard of care) for the treatment of adult subjects with chronic immune thrombocytopenia (idiopathic…
The primary objective of this study is to compare overall survival (OS) in patients with progressive PSMA-positive mCRPC who receive 177Lu-PSMA-617 in addition to best supportive/best standard of care versus patients treated with best supportive/…
To assess the ability of MRI techniques to detect early stages of lipid accumulation in the liver of ASMD patients with the chronic visceral subtype compared to values of age-, sex- and BMI-matched healthy subjects.