Alexion Pharmaceuticals, Inc.

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already receiving standard treatments but still experience significant red blood cell destruction. Participants must be on SOLIRIS or ULTOMIRIS for at least 6 months …

This study is testing whether the drug ravulizumab can help children and teenagers (ages 2-18) with IgA nephropathy, a kidney disease where the immune system attacks the kidneys. Participants will receive the drug through an IV to see if it reduces protein in their urine and slow…

This study is testing whether the drug Ravulizumab can help control symptoms and improve daily life for people with early-stage myasthenia gravis, a condition that causes muscle weakness. Researchers will follow 40 participants for about a year to see how well the drug works in r…

This study is testing a new injectable drug, ALXN2420, to see if it works better than a placebo when added to standard acromegaly medications. It will involve about 60 adults whose current treatment isn't fully controlling their hormone levels. The main goal is to see if the new …

This study is testing an investigational drug called tarperprumig for adults with ANCA-associated vasculitis, a rare autoimmune disease that causes blood vessel inflammation. The main goal is to see if the drug is safe and can help patients achieve and maintain disease remission …

This study is testing a medication called ravulizumab to see if it can safely prevent relapses in children and adolescents with a rare autoimmune disease called NMOSD. The disease attacks the nerves in the eyes and spinal cord. Researchers will compare the relapse rate in partici…

This study is testing an investigational drug called ALXN1920 to see if it can reduce harmful protein loss in the urine of adults with primary membranous nephropathy, a serious autoimmune kidney disease. About 30 participants at high risk for their disease getting worse will rece…

This study aims to collect long-term, real-world data on how well specific C5 inhibitor medications control relapses and affect quality of life for adults with AQP4+ NMOSD, a rare autoimmune disorder that attacks the nerves in the eyes and spinal cord. It will follow 122 particip…

This study is testing an investigational drug called gefurulimab in children and teenagers (ages 6 to under 18) who have generalized myasthenia gravis (gMG), a condition that causes severe muscle weakness. The main goals are to see how the drug behaves in the body, if it is safe,…

This study is testing how well the drug ravulizumab works to control atypical hemolytic uremic syndrome (aHUS), a rare disease where the immune system attacks blood cells and damages kidneys. The research will follow 20 participants for 26 weeks to see if the treatment improves t…

This study is testing whether adding a new drug called danicopan helps children with a rare blood disease called PNH. The children are already on standard treatment but still have significant anemia. The main goal is to see if the add-on drug increases their hemoglobin levels (a …

This study is testing whether the drug ravulizumab can slow the progression of IgA nephropathy (IgAN), a kidney disease that can lead to kidney failure. About 510 adults with IgAN who are at high risk of their disease getting worse will receive either the study drug or a placebo …

This study is checking if the drug acoramidis can slow down the progression of a serious heart condition called transthyretin cardiac amyloidosis, where abnormal proteins build up in the heart. It will follow 200 patients who are either newly starting the drug or switching from a…

This study is testing whether an intravenous medication called ravulizumab can help a newly transplanted kidney start working properly sooner in patients at high risk for complications. It will involve 450 adults receiving a kidney from a deceased donor who are likely to experien…

This study is testing whether an experimental drug called ALXN2030 can help control antibody-mediated rejection in adults who have received kidney transplants. About 45 participants will receive either one of two doses of ALXN2030 or a placebo for 52 weeks, along with their stand…

This early-stage study is testing a new, one-time gene therapy called ALXN2350 for adults with a specific genetic form of heart muscle disease (BAG3 mutation). The main goals are to find a safe dose and see if the treatment helps control the disease. It will involve a small group…

This study is testing a new type of personalized cell therapy called AZD0120 in people with AL amyloidosis that has come back or hasn't responded to prior treatments. The therapy involves modifying a patient's own immune cells to target and destroy the harmful cells causing the d…

This is an observational registry study that will follow 3,000 patients diagnosed with atypical Hemolytic-Uremic Syndrome (aHUS), a rare and serious blood disorder. The study aims to collect long-term safety information on patients who are treated with specific medications (eculi…

This study aims to understand if the immune system of patients with a rare bone disease, hypophosphatasia, can stop their main treatment, asfotase alfa, from working or cause severe allergic reactions. It will follow about 30 patients, including children and adults, for at least …

This study is creating a worldwide registry to collect long-term health information from people diagnosed with Lysosomal Acid Lipase (LAL) Deficiency, a rare genetic disorder that causes severe liver and heart problems. By tracking up to 300 patients over time, researchers aim to…

This study is creating a worldwide registry to track 500 patients with generalized myasthenia gravis who have received specific antibody treatments. Researchers will collect health information over up to 5 years to understand how these treatments affect patients' daily lives and …

This study aims to gather information on the safety of the drug Ultomiris (ravulizumab) during pregnancy and breastfeeding. It will follow about 75 women who have rare conditions like PNH or aHUS and who took Ultomiris while pregnant. Researchers will track the health of both the…