Alexion Pharmaceuticals, Inc.

This study tests ravulizumab, a drug that calms a part of the immune system, in 12 children with NMOSD – a rare disease where the immune system attacks the nerves in the eyes and spine. The goal is to see if the drug can reduce the number of relapses and prevent disability over a…

This phase 3 trial tests ravulizumab, a drug that blocks part of the immune system, against a placebo in 579 adults with IgA nephropathy—a kidney disease that can lead to kidney failure. The goal is to see if ravulizumab reduces protein in the urine and preserves kidney function …

This phase 3 trial tests an experimental drug called ALXN1850 in 124 adolescents and adults with hypophosphatasia, a rare genetic bone disease. Participants receive either the drug or a placebo by injection under the skin. The main goal is to see if the drug improves walking dist…

This phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell diso…

This phase 3 trial tests whether ALXN1720 can improve daily living and muscle strength in adults with generalized myasthenia gravis who have specific antibodies. About 260 participants will receive either the drug or a placebo for 26 weeks. The study is active but no longer recru…

This study follows about 80 people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken danicopan in a previous trial. They will continue taking danicopan as an add-on to their standard C5 inhibitor therapy. The goal is to see if the combination remains safe and …

This study tests a new drug called ALXN1850 in children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. The trial compares the drug to a placebo to see if it improves bone health and movement. About 30 children who have not received prior treatmen…

This study compares a new medicine, ALXN1850, to 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. T…

This study tests a drug called ravulizumab in 12 children aged 6 to 18 with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. The goal is to see how the drug works in the body and if it helps control symptoms. Participants receive the drug through an I…

This study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live …

This Phase 3 trial tests an experimental drug called ALXN2220 in about 1,180 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart, causing heart failure. The drug is designed to remove those protein deposits. Partic…

This study follows 50 adults with PNH who take danicopan along with their usual medication (eculizumab or ravulizumab). Researchers track serious side effects and infections over time to see if the combination is safe for long-term use. Participants are already in a PNH registry …

This study looks at the long-term safety of danicopan when added to standard treatments (Soliris or Ultomiris) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will analyze data from 50 adult patients in an international registry, trac…

This study follows 4 people with methylmalonic acidemia who previously received hLB-001 gene therapy. Researchers are checking for long-term side effects. The goal is to see if the treatment remains safe over time.

This study planned to test a combination of immunosuppressive drugs (methotrexate, rituximab, bortezomib, IVIg, and folic acid) in people with hypophosphatasia whose bodies had stopped responding to asfotase alfa due to antibodies. The goal was to see if suppressing the immune sy…

This study tests a drug called eculizumab in Chinese adults with a rare autoimmune disease called NMOSD, which attacks the nerves in the eyes and spinal cord. The goal is to see if the drug can reduce the number of relapses (flare-ups) and slow disability. Participants must have …

This phase 3 trial tests eneboparatide, a lab-made hormone, in 165 adults with chronic hypoparathyroidism. The goal is to see if it can help patients stop taking high-dose calcium and vitamin D supplements while keeping calcium levels normal. Participants receive either the drug …

This study follows about 35 people with dilated cardiomyopathy caused by a BAG3 gene mutation, a condition that weakens the heart muscle. Over three years, researchers will monitor heart function, symptoms, and quality of life using tests like imaging and blood work. The goal is …

This early-stage study tests the safety of a new drug called ALXN2030 in 48 healthy adults. Participants receive a single injection of the drug or a placebo, and researchers monitor for side effects, how the drug moves through the body, and its effect on the immune system. The go…

This long-term study follows over 1,500 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 long-term safety and effectiveness of the treatment asfotase alfa. Th…