3 results
Approved WMOCompleted
To assess the occurrence of lung shunting and to evaluate the biodistribution and safety of Technetium 99m labelled macroaggregated albumin (Tc-99m-MAA) after administration to the kidney.
Approved WMOPending
Primary objective of the study is to determine the ability of ITF2357, administered orally at the dose of 50 mg b.i.d. for 8 consecutive weeks, to induce complete healing of mucosal ulcerations of ileum and/or colon, assessed by endoscopy,Secondary…
Approved WMORecruiting
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…