5 results
Primary Objectives:* For the European Union (EU) regulatory assessment, the primary objective of Period 1 is to evaluate theefficacy of tadalafil compared with placebo, as measured by time to clinical worsening (CW) in pediatricPAH patients through…
The primary objective of this study is to compare the two treatment strategies; first-line combination therapy (ambrisentan and tadalafil) versus first-line monotherapy (ambrisentan or tadalafil) in subjects with PAH. This will be assessed by time…
The primary objective is to assess the proportion of patients in each treatment arm with a satisfactory clinical response as defined by a composite primary endpoint at Week 24.The secondary objective is to demonstrate safety and clinical effect at…
The primary objective is to compare the effectiveness of ambroxol versus placebo on the MDS-UPDRS part III motor sub-score in the *practically defined OFF-medication state* in patients with moderate PD, carrying a GBA mutation. The hypothesis is…
Primary Objective:Part A:To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease progression assessed by reachable workspace quantification of total relative surface area (RSA) Q1-Q5 with 500 g wrist…