Alexion Pharmaceuticals, Inc.

This study tests a drug called ravulizumab in children aged 2 to 18 with a kidney disease called IgA nephropathy. The goal is to see if the drug can reduce protein in the urine, a sign of kidney damage. Participants will receive the drug through an IV and be monitored for safety …

This study tests a new treatment called AZD0120 for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy harmful cells. The study aims to see if it is safe and effectiv…

This phase 3 trial tests whether ravulizumab, given intravenously, can reduce the severity of delayed graft function (DGF) in adults at high risk after kidney transplant. DGF is when the new kidney is slow to start working, often requiring dialysis. The study will compare ravuliz…

This study tests an experimental drug called ALXN1920 in 30 adults with primary membranous nephropathy, a rare kidney disease that can lead to kidney failure. Participants are at high risk for their disease getting worse. The study compares the drug to a placebo to see if it redu…

This study tests a new drug called tarperprumig in 75 adults with ANCA-associated vasculitis, a rare disease where the immune system attacks blood vessels. The goal is to see if the drug is safe and can help control the disease by reducing inflammation. Participants will receive …

This early-stage study tests a one-time gene therapy called ALXN2350 for adults with a specific genetic heart condition (BAG3-related dilated cardiomyopathy). The heart muscle becomes weak and enlarged, making it hard to pump blood. The treatment aims to fix the faulty gene to im…

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, an…

This study is testing the drug ravulizumab in 20 people with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. The goal is to see if the drug improves platelet counts and kidney function over 26 weeks. All participants receive …

This study is testing whether the drug ravulizumab can help people with generalized myasthenia gravis (gMG) safely reduce or stop taking oral steroids. About 75 adults who are already on ravulizumab and taking at least 7.5 mg of prednisone daily will follow a gradual steroid-tape…

This study tests a drug called gefurulimab in 12 children aged 6 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The drug works by blocking a part of the immune system to reduce symptoms. Participants receive weekly injections, and researchers w…

This study tests whether the drug ravulizumab can improve daily activities in people with early-stage generalized myasthenia gravis who have a specific antibody (AChR-positive). About 40 participants will receive treatment and be followed for 50 weeks. The main goal is to see if …

This study tests a medication called acoramidis in 200 people with a rare heart condition called transthyretin cardiac amyloidosis. The goal is to see if the drug can slow down the disease and prevent worsening of heart function. Participants either are newly diagnosed or switch …

This phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participa…

This study is a long-term registry for adults with AQP4+ neuromyelitis optica spectrum disorder (NMOSD) who are already taking Alexion C5 inhibitor drugs like eculizumab or ravulizumab. It will collect information on relapses, safety, and quality of life over many years to unders…

This study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This study tests a single dose of ALXN2230 in 48 healthy adults to see if it is safe and how the body processes it. Participants receive either the drug or a placebo by injection. The goal is to check for side effects and measure drug levels in the blood.

This study is a registry that will follow up to 300 people with LAL deficiency, a rare genetic disorder that causes fat buildup in the body. Researchers will collect information over time to better understand how the disease progresses and how it affects patients. No new treatmen…

This study is a long-term, global registry that follows 500 people with generalized myasthenia gravis who are taking or have taken C5 inhibitor drugs like Soliris or Ultomiris. The goal is to collect real-world data on safety and how well the treatment helps with daily activities…

This study follows 30 people with hypophosphatasia (HPP) who are taking the drug asfotase alfa. Researchers want to see if the immune system can make the drug less effective or cause serious allergic reactions. Participants will be monitored for at least 5 years to track these ri…

This observational study follows 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare and serious kidney condition. Researchers will collect safety and effectiveness data on patients treated with eculizumab or ravulizumab, as well as those receiving other care. The…