Alexion Pharmaceuticals, Inc.

This study tests a new medicine called ALXN1850 for people aged 12 and older with hypophosphatasia (HPP), a rare bone disease. Participants receive either the drug or a placebo to see if it improves walking, strength, and daily function. The trial involves 124 people across multi…

This study tests a new medicine called ALXN1850 in children aged 2 to 12 who have hypophosphatasia (HPP), a rare genetic condition that weakens bones. All participants have already been on the current treatment, asfotase alfa, for at least 6 months. The goal is to see if ALXN1850…

This study tests a new drug called ALXN1720 in adults with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. The goal is to see if the drug improves daily activities and muscle strength compared to a placebo. About 261 participants with specific antibo…

This study tests an experimental drug called CAEL-101 in people with AL amyloidosis, a rare disease where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove these protein deposits. The trial includes 281 participants who have not recei…

This study tests eculizumab in 21 Chinese adults with a severe autoimmune disease called NMOSD that attacks the nerves. The goal is to see if the drug can reduce the number of relapses and slow disability. Participants must already be on stable immunosuppressant medication, which…

This study looks at the long-term safety and effectiveness of danicopan when added to standard treatment for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 80 adults who have already taken danicopan in a previous study will continue treatment.…

This study tests a drug called ravulizumab in children aged 6 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to see how the drug works in the body and if it helps control symptoms. Twelve participants will receive the drug through a…

This study tests an experimental drug called ALXN2220 in adults with a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM), where abnormal proteins build up in the heart and make it weak. About 1,181 participants will receive either the drug or a placebo to see …

This study aimed to see if adding immunosuppressive therapy could help people with hypophosphatasia who stopped responding to the drug Strensiq due to their immune system. The study was withdrawn before enrolling any participants. It focused on patients who had antibodies against…

This study looks at the long-term safety of danicopan when added to standard treatments for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will analyze data from 50 adults already taking danicopan alongside eculizumab or ravulizumab. The goal is to …

This study tests an experimental drug called CAEL-101 in people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug aims to remove these protein deposits to help patients live longer and avoid heart-related ho…

This study tests if a single dose of ravulizumab can prevent serious kidney problems after heart surgery in adults with chronic kidney disease (CKD). About 736 participants will receive either the drug or a placebo before surgery. The goal is to see if the drug reduces the risk o…

This study looks at the long-term safety of adding danicopan to standard treatments (ravulizumab or eculizumab) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will analyze data from 50 adults already enrolled in a PNH registry to tra…

This study tests a new medicine called ALXN1850 in children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. The goal is to see if the drug improves bone healing and movement compared to a placebo. About 30 children who have not received prior trea…

This study tests a new drug called eneboparatide for people with chronic hypoparathyroidism, a condition where the body doesn't make enough parathyroid hormone. The goal is to see if the drug can help patients stop or reduce their daily calcium and vitamin D supplements while kee…

This study follows 4 people with methylmalonic acidemia (MMA) who previously received the experimental gene therapy hLB-001. Researchers are checking for any long-term side effects or health problems that may appear years after treatment. The goal is to understand the lasting saf…

This study follows about 35 people with a specific genetic heart condition called BAG3-related dilated cardiomyopathy, where the heart is enlarged and weak. Researchers will monitor participants for three years using heart scans, blood tests, and activity tracking to understand h…

This early-stage study tests whether a single dose of the experimental drug ALXN2030 is safe and tolerable in healthy adults. About 48 participants will receive either the drug or a placebo. The goal is to gather safety data and understand how the drug moves through the body, not…

This study is a long-term registry that follows people of all ages with hypophosphatasia (HPP), a rare bone disease. Researchers will collect information on how the disease progresses, its impact on daily life, and the safety and effectiveness of the treatment asfotase alfa. The …