Alexion Pharmaceuticals, Inc.

This study tests a single dose of ravulizumab given before heart surgery to see if it can prevent kidney damage and serious kidney problems in people with chronic kidney disease. About 736 adults will take part. The goal is to reduce the risk of major kidney events within 90 days…

This study tested a drug called ravulizumab in people aged 12 and older who developed a serious blood clotting condition (thrombotic microangiopathy) after a stem cell transplant. The goal was to see if the drug could prevent death or worsening of the condition over 26 weeks. Par…

This study tested a drug called ALXN2060 (acoramidis) in 25 Japanese patients with a rare heart condition called ATTR-CM, which causes heart failure. The drug is taken as a tablet twice daily. Researchers measured how far patients could walk in 6 minutes and tracked heart-related…

This completed Phase 3 trial tested ravulizumab in 18 adults in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study measured changes in LDH, a marker of red blood cell breakdown, to see if the drug could control the…

This study tested eculizumab in 25 adults with PNH in China who had not previously taken complement inhibitors. Participants received infusions over 62 weeks. The main goal was to see if the drug reduced LDH levels, a marker of disease activity. Results showed a decrease in LDH, …

This study followed 120 adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from the older drug eculizumab to the newer ravulizumab. Researchers measured changes in a blood marker called LDH to see if the new drug controlled red blood cell breakdown just as well or…

This completed Phase 3 study tested eculizumab in 15 Chinese patients with refractory generalized myasthenia gravis (gMG), a condition where standard treatments fail. Eculizumab is given by IV infusion and aims to improve daily living activities and muscle strength. The study mea…

This study tested a drug called ravulizumab in 41 children who developed a serious blood clotting problem (TMA) after a stem cell transplant. The goal was to see if the drug could help control the condition and improve blood counts and kidney function. The treatment lasted 26 wee…

This study tested the drug eculizumab in 25 children and adults in China with a rare kidney condition called atypical hemolytic uremic syndrome (aHUS). The goal was to see if the drug could stop blood clots and improve kidney function. All participants received eculizumab intrave…

This early study tested a drug called bertilimumab in 11 adults aged 60 and older with newly diagnosed bullous pemphigoid, a condition that causes painful blisters. The main goals were to check safety and see if the drug helps control the disease. While results may show some bene…

This Phase 3 trial tested the drug ravulizumab (Ultomiris) in 58 adults with neuromyelitis optica spectrum disorder (NMOSD), a rare condition where the immune system attacks the optic nerves and spinal cord. The goal was to see if the drug could prevent relapses. Participants rec…

This early-stage trial tested a synthetic parathyroid hormone analog called AZP-3601 in healthy volunteers and patients with chronic hypoparathyroidism, a condition where the body doesn't produce enough parathyroid hormone. The study aimed to check safety and how the drug works i…

This Phase 3 trial tested the drug eculizumab in 57 people with severe Guillain-Barré syndrome (GBS), a rare nerve disorder that can cause paralysis. Participants received either eculizumab or a placebo once a week for four weeks, alongside standard treatment. The study measured …

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This early-stage study tested how a high-fat, high-calorie meal affects the way the body processes acoramidis, a drug being developed for a rare heart condition. Eighteen healthy adults took a single dose of the drug either with or without food. The goal was to see if food change…

This study collected health information from over 6,000 people with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The goal was to track the safety of two medications, Soliris and Ultomiris, and to understand how PNH changes over time. Participants were followe…