8 results
To determine the ratio of parent drug to metabolites in the circulation.Profiling of [14C]-etripamil metabolites in blood, urine and feces.To determine the mass balance of drug-related materials following intranasal administration.To determine the…
The primary objective of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, andefficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).
PrimaryTo assess the efficacy of ravulizumab in the treatment of participants with TMASecondaryTo characterize TMA responseTo assess impact on hemoglobin levels To evaluate change in kidney function To assess duration of Complete TMA Response and…
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 primary objective of this study is to demonstrate the superiority of etripamil NS over placebo in reducing ventricular rate in patients with AF.
Part 1 - To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to…
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
Cohort-Expansion Stage (Single-Agent and Combination Therapy Cohorts):Primary:• To evaluate the preliminary efficacy of XB002 when administered alone and in combination therapy by determining the ORR per RECIST 1.1 (or other applicable response…