3 results
Approved WMOWill not start
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
Approved WMOCompleted
Primary objective:To assess pharmacokinetic similarity of EBR/GZR as a crushed (test) Zepatier tablet compared to a whole (reference) tablet.Secondary objective:To assess Cmax similarity of EBR/GZR as a crushed (test) Zepatier tablet compared to a…
Approved WMORecruiting
The main objective of this study is to determine whether lowering estrogen and progesterone levels with leuprorelin decreases liver growth rates in pre-menopausal women with severe PLD.Secondary objectives are to assess in these women the effect of…