Clinical Research Directory

Ocular Manifestations of Granulomatosis With Polyangiitis.

The current state of knowledge on ANCA-associated vasculitis (AAV) indicates that it is a group of autoimmune diseases in which small blood vessels in various organs are affected. Disease entities included in this group are granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis (EGPA, Churg-Strauss syndrome). These are rare diseases, with an incidence in Europe of approximately 20-25 cases per million people per year. There is a slight predominance among men, and the risk of developing the disease increases with age. ANCA antibodies play a role in the pathogenesis of the disease, and inflammation within small vessels leads to damage of the vessel walls, resulting either in rupture or occlusion of the vessel lumen. Consequently, vital organs such as the kidneys, lungs, heart, nervous system, upper respiratory tract, gastrointestinal tract, and eyes may be affected. If the disease is not diagnosed, untreated, or treated improperly, it can lead to irreversible failure of these organs and even death. Despite appropriate treatment, AAV diseases tend to relapse; therefore, therapy consists of two phases: induction therapy and maintenance therapy. Current EULAR/EDTA guidelines for induction treatment of AAV recommend the use of cyclophosphamide (CYC) or rituximab (RTX) in combination with glucocorticosteroids in cases of severe disease. If remission is achieved after induction therapy, maintenance treatment should be initiated with drugs such as azathioprine, mycophenolate mofetil, methotrexate, or rituximab, combined with a low dose of glucocorticosteroids. Maintenance therapy should last no less than two years. The study will focus on ophthalmological evaluation of patients diagnosed with ANCA-associated vasculitis. In this disease, all structures of the eye may be involved. The most common ocular manifestations include scleritis, keratitis, proptosis, inflammation of orbital tissues, nasolacrimal duct obstruction, and orbital involvement leading to proptosis, double vision, and restricted eye movement. Until recently, the disease was often fatal. However, advances in diagnostics and current pharmacological treatment options, combined with appropriately aggressive immunosuppressive therapy, have significantly improved survival, enhanced patients' quality of life, and reduced mortality. Early diagnosis and prompt initiation of appropriate therapy are crucial.

Efficacy and Safety of Rituximab in the Treatment of Good Prognosis Microscopic Polyangiitis

The purpose of the study is to determine wether a rituximab-based treatment compared to standard therapy (glucocorticoid alone) in patients with microscopic polyangiitis without any bad prognosis marker increases the remission and reduces the relapse free survival rate.

Comparison of a Strategy Based on Clinico-biological Monitoring Versus Pre-emptive Rituximab Treatment in Cases of ANCA Reappearance in Granulomatosis With Polyangiitis and Microscopic Polyangiitis.

The PREP-ANCA study seeks to establish a more personalized treatment strategy for ANCA-associated vasculitides by assessing the efficacy of pre-emptive rituximab administration upon ANCA repositivity in preventing relapses in granulomatosis with polyangiitis and microscopic polyangiitis.

Safe Effective Therapy With Low-Dose Glucocorticoid in ANCA-Associated Vasculitis (SAFE-LOW)

The purpose of this study is to determine the safety and efficacy of a therapeutic regimen consisting of 4 weeks of glucocorticoids given with a combination of the usual induction agents for ANCA-associated vasculitis. The trial will compare this regimen to the current standard of care treatment and glucocorticoid dosing for ANCA-associated vasculitis with severe kidney involvement. This trial will begin as a pilot to assess feasibility of recruitment and of adherence to the intervention.

Vasculitis Pregnancy Registry

The purpose of this study is to learn about the experience of women with vasculitis who become pregnant. In particular, the study will consist of several online surveys to assess 1. each woman's vasculitis severity and pregnancy-related experiences, and 2. pregnancy outcomes.

Clinical Transcriptomics in Systemic Vasculitis (CUTIS)

Multi-center observational study to evaluate the histopathology and transcriptome of cutaneous lesions in patients with several different types of vasculitis.

Special Drug Use-results Survey for Long-term Use（Avacopan）

The purpose of this survey is evaluating the safety and efficacy of long-term administration of avacopan for Japanese patients with microscopic polyangiitis or granulomatosis with polyangiitis under actual conditions of use.