8 results
The primary objective of this study is to determine whether ACE inhibition results in an improvement of microvascular glycocalyx-thickness in patients with type 1 diabetes. The secondary objectives are to investigate whether this (hypothesized)…
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…
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
Primary ObjectiveTo determine if the humoral immune response of a single compared to a two dose COVID-19 vaccination regimen is non-inferior in paediatric subjects who are immunologically primed by natural infection. Secondary Objective(s) 1.To…
To examine the impact of medication induced systolic BP (SBP) reductions >=10 mmHg, while reaching a treatment target of <=140 mmHg, on cerebral blood flow (CBF) in frail elderly with untreated or uncontrolled systolic hypertension at…
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…
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).