3 results
Approved WMOCompleted
Primary Objectives: To evaluate the overall bowel cleansing efficacy and the *Excellent plus Good* cleansing rate in the colon ascendens of a 1-day day before-only split-dosing regimen with NER1006 compared to a 1-day day before-only split-dosing…
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
The primary aim of the trial is to demonstrate the non-inferiority of CSL222 (formerly AMT-061) (2 × 1013 gc/kg) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (CSL222 (formerly AMT-061))…