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Last Updated: 10/31/2025
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney Transplantation
Summary: The primary objective of this study is to demonstrate the efficacy of ravulizumab vs placebo in reducing the severity of DGF as measured by time to freedom from dialysis in adult participants who are at high risk of DGF after undergoing transplant of deceased donor kidney.
Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis (gMG) Naive to Complement Inhibitors. Prospective, Multicenter, Non-interventional Study (MG-ARCADIA).
Summary: The Polish multicentre observational (non-interventional) study aiming to collect data on the management and clinical outcomes of patients with gMG that received ravulizumab.
A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy
Summary: This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizum...
Observational Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Summary: The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Immunoglobulin A Nephropathy (IgAN)
Summary: The primary objective of this study to evaluate efficacy of ravulizumab compared with placebo on proteinuria reduction and change in eGFR in adult participants with IgAN who are at risk of disease progression.
MG-EVOLUTION. Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis: A Real-World Study With Biomarker Analysis
Summary: The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.
An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
Summary: Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.
Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.
Summary: The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
Summary: The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US
Summary: The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to und...
A Phase 3, Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Ravulizumab in Pediatric Participants (2 to < 18 Years of Age) With Primary Immunoglobulin A Nephropathy (IgAN)
Summary: The primary objective of this study is to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population.
Clinical and Radiological Outcomes in People With Aquaporin-4 IgG Positive Neuromyelitis Optica Spectrum Disorder Treated With Ravulizumab
Summary: This is an observational study to: * evaluate the on-treatment clinical performance of ravulizumab in relation to the pre-treatment time period (time period prior to exposure), * enhance knowledge regarding conventional MRI outcomes in people with NMOSD treated with ravulizumab, * identify factors suggestive of subclinical disease progression through conventional MRI sequences, * determine if trea...
Last Updated: 10/31/2025