2 results
Approved WMOCompleted
• To evaluate the tolerability and safety of ascending single oral doses of ACT-541468 in healthy male subjects.• To investigate the single oral dose pharmacokinetic (PK) and PD of ACT-541468 in healthy male subjects.• To investigate dose…
Approved WMOCompleted
Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…