3 results
Primary objectivesPart A of the study (if applicable):1. To assess the safety and tolerability of tosedostat added to standard induction chemotherapy for AMLand select the feasible dose level for part B of the study2. To assess in a randomized…
Primary:• To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU)• To evaluate changes from baseline in dietary phenylalanine (Phe)/protein consumptionSecondary:• To evaluate PTC923 effect on quality of life (QOL) using the…
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…