Alexion Pharmaceuticals, Inc.

This study is testing if adding an investigational drug called CAEL-101 to standard treatment helps people with a severe stage of AL amyloidosis. AL amyloidosis is a disease where abnormal proteins build up and damage organs, especially the heart. The main goal is to see if the c…

This study is testing whether ravulizumab, an intravenous medication, can help control symptoms of generalized myasthenia gravis in children aged 6 to 18. Researchers will measure how the drug works in the body and whether it improves muscle strength and daily activities. The stu…

This study follows patients with a rare blood disease called PNH for up to three years. It tests the long-term safety and effectiveness of adding a drug called danicopan to their standard treatment. The goal is to see if this combination helps control the disease better over time…

This study is testing whether a single dose of the drug ravulizumab can protect the kidneys of people with chronic kidney disease who are having planned heart surgery. The goal is to prevent serious kidney injury and related complications that can occur after surgery. About 736 p…

This study is testing whether an experimental drug called ALXN1850 can improve bone health and physical abilities in children aged 2-12 with hypophosphatasia, a rare genetic condition that weakens bones. Participants receive either the drug or a placebo through injections under t…

This study is testing whether a daily injection called eneboparatide can help people with chronic hypoparathyroidism. For the first 24 weeks, 165 participants will receive either the real injection or a placebo (dummy injection), with neither patients nor doctors knowing which. T…

This study is testing a new drug called ALXN1850 against the current standard treatment (asfotase alfa) in children aged 2 to under 12 who have a rare bone-weakening condition called hypophosphatasia (HPP). The main goal is to see if the new drug is as safe and well-tolerated as …

This study is testing a drug called eculizumab in Chinese adults with a rare autoimmune disease called Neuromyelitis Optica Spectrum Disorder (NMOSD). The main goal is to see if the drug can reduce the frequency of disease attacks (relapses) and slow disability progression. Resea…

This study is observing the long-term safety of adding a medication called danicopan to standard treatments for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. It will follow about 50 patients already taking this combination therapy to monitor for ser…

This study aims to understand the long-term safety of adding the drug danicopan to standard treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. Researchers will analyze existing health data from about 50 adult patients already taking these medications. …

This study is testing whether an investigational drug called ALXN1720 is safe and effective for adults with generalized myasthenia gravis, a chronic autoimmune disease that causes severe muscle weakness. About 261 participants will receive either the drug or a placebo for 26 week…

This study aimed to help patients with hypophosphatasia, a rare bone disorder, who started responding to their main treatment (asfotase alfa) but then lost that benefit. Researchers planned to test if adding immune-suppressing drugs could restore the treatment's effectiveness in …

This study is monitoring the long-term safety of four patients who previously received an experimental gene therapy (hLB-001) for a rare genetic disorder called methylmalonic acidemia (MMA). The main goal is to check for any side effects that may appear over a very long period—up…

This large, late-stage study is testing whether the drug ALXN2220 can help adults with a serious heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). The main goal is to see if the drug reduces the combined number of deaths from any cause and serious heart-relat…

This study is testing whether a new drug called ALXN1850 can improve walking ability, strength, and reduce pain in teenagers and adults with hypophosphatasia, a rare genetic bone disorder. Participants who have never taken the existing treatment (asfotase alfa) will receive eithe…

This study is testing whether the drug ravulizumab can help control a dangerous blood clotting and organ damage condition (TMA) that sometimes occurs after a stem cell transplant. It will compare the drug plus standard care against a placebo plus standard care in about 148 partic…

This study is testing whether adding an experimental antibody called CAEL-101 to standard treatment helps people with a serious stage of AL amyloidosis, a disease where abnormal proteins build up and damage organs like the heart. The antibody is designed to help clear these harmf…

This early-stage study aims to check the safety and side effects of a single dose of an experimental drug called ALXN2030 in healthy people. It will involve 48 healthy adult volunteers, with some receiving the drug and others receiving a placebo. Researchers will measure how the …

This study aims to understand how a specific genetic heart condition (BAG3 dilated cardiomyopathy) naturally progresses over three years. Researchers will observe 35 participants who have this genetic mutation and reduced heart function, while they continue their usual treatments…

This study is creating a long-term registry to better understand hypophosphatasia (HPP), a rare genetic bone disorder. It will follow over 1,500 patients of all ages worldwide to track how the disease progresses naturally and how it affects daily life. The registry will also moni…