Alexion Pharmaceuticals, Inc.

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 a new drug called tarperprumig for people with ANCA-associated vasculitis, a rare disease where the immune system attacks blood vessels. The trial will include 75 adults with newly diagnosed or relapsing disease. Researchers aim to see if the drug is safe and can…

This study tests an experimental drug called ALXN1920 in 30 adults with primary membranous nephropathy, a 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 reduces p…

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 and improve heart health markers. Participants are either newly diagnosed or switching …

This study tests a drug called gefurulimab in 12 children aged 6 to 18 who have generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to see how the drug moves through the body, how well it works, and if it is safe. Participants will receive the drug…

This early-stage study tests a single-dose gene therapy called ALXN2350 in 6 adults with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if the treatment is safe and can improve heart function. Participants must have stable heart failure treatment and no…

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+). About 40 participants will receive treatment and be followed for 50 weeks. The goal is to see if starting trea…

This study looks at whether people with generalized myasthenia gravis (gMG) who are already taking the drug ravulizumab can safely reduce or stop their oral steroids. About 75 adults will follow a step-by-step steroid-tapering schedule. The goal is to lower steroid doses without …

This phase 3 study tests whether ravulizumab can reduce protein in the 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 sl…

This study tests a medicine called ravulizumab in children with neuromyelitis optica spectrum disorder (NMOSD), a rare disease where the immune system attacks the nerves. The goal is to see if the drug can reduce the number of relapses (flare-ups) and slow disability. About 12 ch…

This study tests a new treatment called AZD0120 for people with AL amyloidosis that has come back or not responded to prior therapy. The treatment uses a patient's own immune cells, modified to target and destroy harmful cells. The goal is to see if it is safe and can reduce or e…

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 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, which is a sign of kidney damage. Participants receive the drug through an IV and are monitored for sa…

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant anemia despite current treatment with Soliris or Ultomiris. Participants must be at least 18 years old, have been on Soliris or Ultomiris for 6 months, a…

This study tests if the drug ravulizumab can help transplanted kidneys start working sooner, reducing the need for dialysis. It involves 450 adults at high risk for delayed graft function after receiving a deceased donor kidney. Participants will receive either ravulizumab or a p…

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 is a long-term registry that will follow about 500 people with generalized myasthenia gravis (gMG) who are taking or have taken C5 inhibitor drugs like Soliris or Ultomiris. Researchers will collect information on safety and how well the drugs help with daily activitie…

This study is a long-term registry for adults with AQP4+ neuromyelitis optica spectrum disorder (NMOSD) who are already taking Alexion C5 inhibitor drugs (eculizumab or ravulizumab) to prevent relapses. Researchers will collect data on relapse rates, safety, and quality of life o…

This study looks at the safety of the drug Ultomiris when used during pregnancy. Researchers will track 75 pregnant or postpartum women who take Ultomiris for conditions like PNH or aHUS. They will record pregnancy complications, maternal health issues, and infant health outcomes…

This study follows 30 people with hypophosphatasia (HPP) who are taking or starting 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 monitored for at least 5 years.

This study is a registry that collects information from people with LAL deficiency, a rare genetic disorder. It does not provide any treatment but aims to better understand how the disease progresses and varies among patients. Up to 300 participants will be followed over time to …

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.