3 results
Approved WMOPending
To assess the bile acid composition of cystic bile and serum pharmacokinetics after a 3-week treatment with UDCA and to correlate pharmacokinetic parameter sof UDCA in bile and serum during steady state.To compare the composition of bile acids and…
Approved WMOCompleted
To compare the effect on cognition of treatment with clobazam or corticosteroids.
Approved WMORecruiting
The primary objectives of the study are to:Determine the OBD(s) and RP2D's) of BMF-219 monotherapy administered daily based on evaluation of all available PK/ PD, target engagement, safety, and tolerability data. Note that the OBD and RP2D may…