4 results
Primary Objective:To compare the efficacy of BMS-986142 versus placebo on a background of MTX as assessed by ACR20 and ACR70 response rates at week 12.Secondary Objectives:1) Assess additional efficacy outcomes of BMS-986142 at week 12 and over 12…
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…
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 assess the proportion of patients with intervention failure at 12 months after dose reduction, defined as patients who have restarted their initial dose due to (expected) loss of major molecular response.