Efficacy and Safety of Benralizumab in EGPA Compared to Mepolizumab.
Eosinophilic Granulomatous VasculitisThis is a randomized, double blind, active-controlled, parallel group, multicenter 52-week Phase 3 study to compare the efficacy and safety of benralizumab 30 mg versus mepolizumab 300 mg administered by subcutaneous (SC) injection in patients with relapsing or refractory EGPA on corticosteroid therapy with or without stable immunosuppressive therapy. All patients who complete the 52-week double-blind treatment period on IP may be eligible to continue into an open label extension (OLE) period. The OLE period is intended to allow each patient at least 1 year of treatment with open-label benralizumab 30 mg administered SC (earlier enrolled patients may therefore be in the OLE for longer than 1 year).
Biologics in Refractory Vasculitis
Giant Cell ArteritisTakayasu Arteritis7 moreVasculitis occur when the body's immune system, rather than protecting the body, attacks blood vessels, causing injury to the vessel and the part of the body it supplies with blood. Vasculitis is rare, and there are a number of different types, which can affect both adults and children. We treat vasculitis with steroids and drugs aiming to damp down the activity of the immune system, but they often cause side effects. Some patients do not improve with this treatment, or cannot tolerate it and their vasculitis worsens; this is known as refractory vasculitis. Patients with refractory vasculitis are at high risk of health complications from the disease and its therapy and are in need of newer more effective treatments with fewer side effects. Biologics are drugs which are designed to precisely target parts of the immune system and may have fewer side effects. Biologics have been used for several years to treat vasculitis, particularly anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis or AAV. However, for many of the rarer types of vasculitis, and especially those vasculitis disease types that are not ANCA-associated, there is little information to support use of biologic therapies as effective treatments. The purpose of this trial is to find out whether biologics are effective and represent value for money for participants with refractory vasculitis. The trial will include patients with Non-ANCA-associated vasculitis (NAAV)
Biomarkers in Autoimmune Diseases, Vasculitis and Auto Inflammatory Diseases
Autoimmune DiseasesVasculitis1 moreThe objective of this work is to identify, in patients with autoimmune diseases, systemic vasculitis and autoinflammatory disease, cytokine and lymphocyte biomarkers of activity of these diseases to identify follow-up biomarkers, in order to personalize the follow-up and the treatments for each patient. Immunological data will be obtained from biological samples collected as part of the usual patient care pathway (Blood and tissues sampling) The study will take place in the Department of Internal Medicine and Clinical Immunology (DMIIC), that is certified as the National Reference Centre for Rare Systemic Autoimmune Diseases and the National Reference Centre for Inflammatory Autoinflammatory Diseases and Inflammatory Amyloidosis (CEREMAIA). Its objective is to contribute to the advancement of fundamental knowledge in immunology, in particular to develop prognostic biomarkers of the activity of autoimmune diseases, systemic vasculitis and autoinflammatory diseases by using blood tests.
Studies of the Natural History, Pathogenesis, and Outcome of Idiopathic Systemic Vasculitis
Takayasu's ArteritisGiant Cell Arteritis3 moreBackground: - Vasculitis is a group of diseases that inflame and damage blood vessels and tissue. It can cause many medical problems. Few tests can diagnose the disease, and none can reliably predict a relapse. Researchers want to study people s genes and follow people over time to see how the disease affects them. Objective: - To learn the signs, symptoms, imaging tests, genetic markers, and blood tests that can help identify people with vasculitis and predict what will happen to them over time. Eligibility: People age 3 and older who have or are thought to have vasculitis, or are related to someone with it. Healthy volunteers. Design: Participants will be evaluated by a doctor who has expertise caring for patients with vasculitis. Participants will give a blood sample. Some will give a urine sample. Some participants may have brushings or biopsies taken from the inside lining of the nose. Images of participants blood vessels may be taken using scans. For some scans, participants will lie on a table that moves in and out of a cylinder that takes pictures. For some scans, a contrast agent may be injected into an arm vein. Other scans may use a radioactive form of sugar. Healthy minors will not have scans. Some participants will answer questionnaires. - Some participants will have their tests done at NIH. Others will have their doctor take the blood, saliva, or cheek swab samples and send them to NIH. Some participants will have one visit lasting 1-2 (but sometimes up to 4) days. Some participants may have follow-up visits every 3 - 6 months, indefinitely.
Pediatric Vasculitis Initiative
Wegeners Granulomatosis (Granulomatosis With Polyangiitis)Microscopic Polyangiitis5 moreChildhood chronic vasculitis describes a group of rare life-threatening diseases that have in common inflammation of blood vessels in vital organs such as kidneys, lungs and brain. Most knowledge about them comes from adult patients. Severe disease requires aggressive life-saving treatments with steroids and some cancer drugs which can themselves cause damage, and increase risks of cancer and severe infections. Conversely, milder disease can be treated with less toxic drugs. Different classification and "scoring tools" are used to define the types and severity of vasculitis and to measure damage caused by disease or drugs. These in turn help direct how aggressively to treat a patient and to measure outcome. None of these tools however have been assessed in children and the best balance of disease and treatment risks against outcome for children is not known. Although causes of these diseases in children and adults are probably the same, the effects of the disease and the response (good and bad) to drugs will differ in growing children. Because specialists may see only one new child with vasculitis each year, obtaining enough information to learn about childhood vasculitis requires cooperation. We will use an international web-based registry to which doctors from 50 or more centers can contribute patient data. We will determine the features which help better classify and diagnose children compared to adults. Through the web we will collect and analyze information on patients similarly classified and "scored" so that most successful treatments can be identified. Children with vasculitis are less likely to have diseases associated with aging, alcohol and smoking etc., and therefore may be a better group in whom to study the underlying biology of vasculitis. We will use this opportunity and collect spit, blood and tissue from registry patients for laboratory study with an aim to find biomarkers to better classify, define and direct optimal treatment and outcomes.
Swiss Pediatric Inflammatory Brain Disease Registry (Swiss-Ped-IBrainD)
Optic NeuritisTransverse Myelitis22 moreThe Swiss-Ped-IBrainD is a national patient registry that collects information on diagnosis, symptoms, treatment, and follow-up of pediatric patients with an inflammatory brain disease in Switzerland. It was first implemented in 2020 in the pediatric clinic of the university hospital in Bern. Further centers all over Switzerland were opened for recruitment in 2021; Aarau, Basel, Bellinzona, Chur, Geneva, Lausanne, Lucerne, St. Gallen, and Zurich. The center in Winterthur is expected to be open for recruitment by autumn 2021. The registry provides data for national and international monitoring and research. It supports research on inflammatory brain diseases in Switzerland and the exchange of knowledge between clinicians, researchers, and therapists. The registry aims to improve the treatment of children with inflammatory brain diseases and optimizing their health care and quality of life.
National Vaccine Adverse Event Reporting Survey and Etiology
COVID-19Vaccine Adverse Reaction12 moreIf your serious vaccine-induced adverse event has been entered in the CDC Vaccine Adverse Event Reporting System (VAERS) we are interested in enrolling you for this study in order to log your symptoms. The primary goal of this study is to create a national database and gather vaccine-associated serious adverse events/injury data from newly vaccinated individuals in the US in order to identify the possible underlying causal relationships and plausible underlying biological mechanisms. The project aims to identify the genetic determinants of vaccine-induced adverse response by studying host genetics. We plan to use whole genome sequencing to identify single nucleotide polymorphisms associated with cardiovascular, neurological, gastrointestinal, musculoskeletal and immunological symptoms induced by vaccine administration. The secondary goal is to establish criteria that enable classification of vaccine-induced adverse events/injuries compare data from our database with the official Vaccine Injury Table National Vaccine Injury Compensation Program on or after March 21, 2017. The tertiary goal is to establish a database to gather detailed long-term adverse reaction data from subjects enrolled in FDA Emergency Use Authorized vaccine clinical trials.
Multicenter Cohort Study of AAV in Hunan of China
ANCA Associated VasculitisThis study aimed to explore the incidence of Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) progression and its association with adverse consequences. It will enroll approximately 500 AAV patients in Hunan province of China and follow-up for at least 5 years. Demographic characteristics, clinical and laboratory data will be collected at baseline and every follow-up. The principal clinical outcomes of the study consist of end stage renal disease (ESRD) and death.
Cardiovascular Status of Children 5 Years After Kawasaki Disease
VasculitisSystemic1 moreThe aim of present study is to determine cardiovascular status of children who had KD in past and to identify possible biochemical markers of cardiovascular damage in those patients. In this cross-sectional study children with history of KD will be examined 5 years after receiving intravenous immunoglobulin treatment (IVIG) and compared to healthy controls in terms of: serum levels of endothelial injury markers (circulating endothelial cells, endocan, soluble thrombomodulin, vascular endothelial growth factor (VEGF) and soluble E-selectin), peripheral blood pressure, central blood pressure, arterial stiffness parameters (measured by applanation tonometry), carotid intima media thickness (cIMT), capillaroscopy and echocardiography.
Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome
Schnitzler SyndromeUrticarial Vasculitis With Monoclonal Immunoglobulin M Component1 moreThe primary goal of the study is to assess the efficacy and safety of RPH-104 in subjects with Schnitzler Syndrome using Schnitzler Disease Activity Score (SDAS), which includes the Physician's Global Assessment (PGA) and the local laboratory C-reactive protein (CRP) result