9 results
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…
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).
To evaluate the efficacy of ravulizumab compared with placebo to reduceproteinuria in adult participants with LN or IgAN.
The primary objective is to collect high-quality 3D mpUS and histology data, to train and improve the classifier algorithm with the goal of achieving an accurate ultrasound imaging tool for the detection of clinically significant prostate cancer.
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
To determine RD of BLU-263To assess if treatment with BLU-263 improves outcomes compared to placebo + BSC, as assessed using the ISM-SAFTo assess the long-term safety and tolerability of treatment with BLU-263To assess the long-term efficacy of…