6 results
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…
The aim of this study is to determine whether LNP023 is effective and safe for the treatment of PNH. LNP023 is compared to the Standard of Care (SOC) anti-C5 antibody treatment. The primary objectives are to:• Demonstrate superiority of LNP023…
The purpose of this study is to ascertain the efficacy, safety, tolerability and pharmacokineticsof LNP023 over a 24-week treatment period compared with rituximab in subjects with MN.
Primary Objective* To explore the pharmacodynamics of ICVT comprised of digoxin and furosemide (dual agent), digoxin (single agent), furosemide (single agent) in patients with AK. * To evaluate clinical efficacy of ICVT comprised of digoxin and…
To evaluate the dose response relationship of LNP023 on the reduction in proteinuria versus placebo after 90 days of treatment in patients with IgA nephropathy.
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…