Eculizumab
Last Updated: 04/28/2026
Eculizumab Add-On Therapy in the Acute Phase of Neuromyelitis Optica Spectrum Disorder: A Multicenter Prospective Real-World Study
Summary: This project is a multi-center, prospective, real-world cohort study that collects clinical data of Chinese patients with AQP4-positive NMOSD in the acute stage. It comprehensively assesses the clinical outcomes of the patients and aims to compare the clinical efficacy and safety of icoxib as a combined add-on treatment versus simple hormone shock therapy during the acute phase of NMOSD.Using simp...
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...
Modified Zipper Therapy for AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder: A Multicenter Randomized Controlled Trial Study
Summary: Study Title: A National, Multicenter, Randomized Controlled Trial of the Modified Zipper Therapy in AQP4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder (ELITE Study) Brief Summary: The goal of this clinical trial is to evaluate the efficacy and safety of a novel sequential immunomodulation strategy, termed Modified Zipper Therapy, in patients with acute attacks of Aquaporin-4 antibody-po...
Long-Term, Observational, Global Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies (C5ITs)
Summary: Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).
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).
Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2 Trial (EASE-NMO)
Summary: Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) a...
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.
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...
Identification of Clinical, Biological, Cellular and Genetic Markers of Favorable Response Complement Inhibitors Therapy in Patients With Generalized Myasthenia Gravis
Summary: Myasthenia gravis is an autoimmune neurological disease caused by autoantibodies primarily directed against components of the postsynaptic membrane of the neuromuscular junction. Approximately 85% of patients have antibodies directed against the acetylcholine receptor (anti-AChR). Anti-AChR antibodies act through three distinct mechanisms: 1. Activation of the classical complement pathway: Formati...
Therapeutic Orientation Test in Thrombotic Microangiopathy
Summary: Haemolytic and Uraemic Syndrome (HUS) is a serious disease requiring rapid diagnosis and management. The atypical HUS diagnosis has been greatly improved by anti-CS antibody (Eculizumab) wich block alternative complement pathway activation. To rise treatment success, Eculizumab introduction should be as early as possible. In some secondary HUS (infection, drugs…) complement is also involved as sec...
A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy
Summary: The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
Eculizumab in Hypertensive Emergency-associated Hemolytic Uremic Syndrome: a Randomized Multicenter Controlled Trial
Summary: Hemolytic and uremic syndrome (HUS) is a clinic-biological syndrome related to thrombotic microangiopathy affecting predominantly the kidney. Atypical HUS (aHUS) has been historically defined as HUS occurring in the absence of infectious event. The role of complement dysregulation in aHUS pathophysiology has been largely demonstrated, since C genetic rare variants are present in 60-70% aHUS patien...
Last Updated: 04/28/2026