Alexion Pharmaceuticals, Inc.

This study tests an experimental drug called ALXN1850 in 124 teens and adults with hypophosphatasia (HPP), a rare genetic condition that weakens bones. Participants receive either the drug or a placebo to see if it improves walking distance and daily function over about 6 months.…

This study tests a new medicine 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 reduce or stop taking high doses of calcium and vitamin D suppleme…

This study tests a drug called ravulizumab in 12 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, how safe it is, and whether it helps control symptoms like trouble moving or brea…

This study tests an investigational drug called ALXN2220 in about 1181 adults with a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM), where abnormal protein builds up in the heart muscle. The drug is designed to remove that protein buildup. The main goal is …

This study tests a new medicine called ALXN1720 for adults with generalized myasthenia gravis, 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 antibod…

This study was designed for people with hypophosphatasia (a rare bone disease) whose treatment with asfotase alfa stopped working because their immune system attacked it. Researchers planned to give immune-suppressing drugs to see if they could restore the treatment's effect. The…

This study tests a single dose of the drug ravulizumab to see if it can prevent serious kidney problems after heart surgery in people with chronic kidney disease (CKD). About 736 adults with moderate to severe CKD will receive either the drug or a placebo before their planned hea…

This study looks at the long-term safety of adding danicopan to standard treatments (eculizumab or ravulizumab) for adults with PNH, a rare blood disorder. Researchers will track serious side effects and infections over time using registry data from 50 participants. The goal is t…

This study tests a new medicine, ALXN1850, against the current standard treatment (asfotase alfa) in 43 children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. All children have been on the standard treatment for at least 6 months before joining.…

This study looks at the long-term safety and effectiveness of danicopan when taken together with a standard medication for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. About 80 adults who previously took danicopan in another stud…

This study monitors the long-term safety of danicopan when used alongside standard treatments (ravulizumab or eculizumab) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track side effects, infections, and other health issues in …

This study tests a new medicine called ALXN1850 in 30 children aged 2 to 12 who have hypophosphatasia, a rare genetic condition that weakens bones. The drug is given as a shot under the skin and compared to a placebo (fake treatment) to see if it helps heal bone problems. All chi…

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. About 281 participants will receive either CAEL-101 or…

This study tests whether eculizumab can reduce the number of relapses in Chinese adults with a rare autoimmune disease called NMOSD. Participants must have had at least one attack in the past year and test positive for a specific antibody. The drug is given alongside stable maint…

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 early-stage study is testing a single dose of a new drug called ALXN2030 in 48 healthy adults. The main goal is to see if it is safe and what side effects it may cause. Researchers will also measure how the drug behaves in the body and how the immune system responds. This is…

This study follows about 35 people with a genetic heart condition called BAG3-related dilated cardiomyopathy, where the heart becomes enlarged and weak. Researchers will track changes in heart function and overall health over three years using tests like imaging, blood work, and …

This study follows 4 patients with methylmalonic acidemia (MMA) who previously received the experimental gene therapy hLB-001. Researchers are tracking any long-term side effects or health problems that may appear over time. The goal is to understand the lasting safety of this tr…

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 …