9 results
This study has two major goals. The first goal of this study is to evaluate whether the disabilitating effects of crowding can be reduced by the use of a magnifier early in life. The second goal is to investigate motor and visual development of…
To evaluate the efficacy of ravulizumab compared with placebo to reduceproteinuria in adult participants with LN or IgAN.
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.
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…
Main objective:To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) profile.Secondary objectives:• To assess the efficacy of…
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).
The primary goal of this study is to evaluate the effectiveness of VR-exposure therapy (VRET) in diminishing anxiety and avoidance within adolescents with social anxiety. We expect that, compared to the within-person baseline and standard treatment…