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, and help control the disease. Participants will receive the drug through an I…

This study looks at whether a planned reduction of oral steroids is safe and effective for adults with generalized myasthenia gravis who are already receiving the drug ravulizumab. About 75 participants will follow a step-by-step tapering schedule. The goal is to see if they can …

This study tests a medicine called ravulizumab in children and teens (ages 2 to 17) with a rare disease called NMOSD that causes inflammation in the brain and spinal cord. The goal is to see if the drug can reduce the number of attacks and slow down disability. Participants recei…

This study tests a drug called gefurulimab in 12 children (ages 6 to 17) who have generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to see how the drug moves through the body, how safe it is, and if it helps improve symptoms. Participants will re…

This study tests a new treatment called AZD0120 for people with AL amyloidosis that has come back or not responded to other therapies. AZD0120 is a type of immunotherapy that uses a patient's own immune cells to target and destroy harmful cells. The study aims to see if this trea…

This study tests an experimental drug called tarperprumig in 75 adults with a rare autoimmune disease that inflames blood vessels (ANCA-associated vasculitis). The goal is to see if the drug is safe and can help control the disease, reducing flare-ups. Participants will receive e…

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 study tests whether the drug ravulizumab can improve daily living 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 goal is to see i…

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite current treatment with Soliris or Ultomiris. Participants must be at least 18 years old, have anemia and high retic…

This study tests an experimental drug, ALXN1920, in 30 adults with primary membranous nephropathy (PMN) who are at high risk for their kidney disease getting worse. Participants will receive either the drug or a placebo, and researchers will measure changes in protein levels in u…

This study tests a drug called acoramidis in 200 people with a rare heart condition called transthyretin cardiac amyloidosis (ATTR-CM). The goal is to see if the drug can slow down the disease by measuring heart stress markers and the need for water pills. Participants either are…

This study tests whether adding danicopan (a pill) to standard PNH therapy (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria (PNH) who still have anemia due to extravascular hemolysis. About 6 participants…

This study tests if the drug ravulizumab can help transplanted kidneys start working sooner, reducing the need for dialysis after surgery. About 450 adults at high risk for delayed graft function will receive either the drug or a placebo. The main goal is to see how quickly patie…

This study tests whether ravulizumab can reduce protein in urine and preserve kidney function in adults with IgA nephropathy, a disease that can lead to kidney failure. About 510 participants will receive either the drug or a placebo. The goal is to see if the drug slows disease …

This study tests a new drug called ALXN2030 in 45 adults who have had a kidney transplant and are experiencing antibody-mediated rejection (AMR), where the body attacks the new kidney. The goal is to see if the drug can help resolve the rejection better than a placebo over 52 wee…

This study tests whether adding a new drug, ALXN2420, to standard acromegaly treatment can better lower a key hormone (IGF-1) linked to the disease. About 60 adults with acromegaly who are already on a stable monthly injection will receive either ALXN2420 or a placebo for 15 week…

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

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

This study is a long-term registry for 500 people with generalized myasthenia gravis (gMG) who are taking or have taken C5 inhibitor therapies like Soliris or Ultomiris. Researchers will collect data on symptoms, daily living activities, and safety over time. The goal is to bette…

This study monitors 75 pregnant or postpartum women who have taken Ultomiris for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and infant health up to one year after birth. The goal is to better understand any risks or complications linked to the drug dur…

This study is a long-term registry that follows about 122 adults with AQP4+ neuromyelitis optica spectrum disorder (NMOSD) who are already prescribed Alexion C5 inhibitor therapies. Researchers will collect data on relapse rates, safety, quality of life, and patient-reported outc…

This study is a registry that collects information from patients with LAL deficiency, a rare genetic disorder. The goal is to better understand how the disease progresses and varies among people. Up to 300 patients will participate, and the data will help improve future care and …

This study is a large, long-term registry that follows up to 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare blood disorder. Researchers will collect safety and health information from both treated and untreated patients to better understand the disease and it…

This early-stage study is testing a new drug, ALXN2230, in 48 healthy adults to see if it is safe and how the body processes it. Participants receive a single injection of either the drug or a placebo. The study tracks side effects, drug levels in the blood, and immune responses.