3 results
Approved WMOCompleted
The objective of this study is to evaluate the possible effect of arginine on galactose oxidative capacity in 5 patients with classic galactosemia (homozygous for the p.Q188R mutation).
Approved WMORecruiting
Objective: To compare pembrolizumab plus paclitaxel with or without bevacizumab to placebo plus paclitaxel with or without bevacizumab, withrespect to progression-free survival (PFS) per RECIST 1.1 as assessed by the investigator.
Approved WMORecruiting
Primary Objective: 1) To evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecundary Objective:1) To evaluate the efficacy of DTX301 in 3 response categories2) To evaluate the effect of…