3 results
Approved WMOPending
The Primary Objective of the study is to dettermine the relative efficacy of AZD2171 [RECENTIN] (both monotherapy or in combination with oral lomustine) compared to oral lomustine alone by assessment of progression free survival (PFS) as assessed by…
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 WMOCompleted
Patients will be evaluated for safety and tolerability of the therapy, but also for signs of clinical efficacy. As long as the patients are on the ICU, measurements of clinical signs and laboratory data will be collected for safety reasons and for…