Disorder of glycosylation

This study tracks 132 people with PNH who are taking Fabhalta to see how safe it is and what side effects occur. Researchers will monitor infections and check for serious red blood cell breakdown if the drug is stopped. The goal is to better understand the drug's risks in everyda…

This study follows about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking pegcetacoplan (Aspaveli/Empaveli) as part of their routine care. Researchers will track blood markers, symptoms, and side effects for up to 3 years to see how well the drug works out…

This phase 3 trial tests crovalimab in about 50 people with PNH who have not used similar drugs before. PNH causes red blood cells to break apart, leading to fatigue, pain, and need for transfusions. Crovalimab is given as an IV infusion and then as injections under the skin to b…

This study tests whether crovalimab works as well as eculizumab for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 210 adults who have not had prior complement inhibitor therapy will receive either drug. The main goals are to see if crovalimab…

This study looks at the long-term safety and tolerability of iptacopan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have already completed earlier phase 2 or 3 studies with the drug. About 208 participants will continue taking iptacopan and be monitored for side e…

This phase 3 trial compares crovalimab, a new injectable drug, to eculizumab in about 190 people with paroxysmal nocturnal hemoglobinuria (PNH) who are already on complement inhibitors. The study aims to see if crovalimab is as safe and effective, with a focus on side effects and…

This study tests the long-term safety of an experimental drug called OMS906 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Participants receive OMS906 as an intravenous infusion every 8 weeks. The goal is to see if this dosing schedule is safe an…

This study monitors about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking iptacopan (Fabhalta). Researchers will use registry data to count infections caused by certain bacteria. The goal is to better understand the real-world safety of this medic…

This study is testing the long-term safety and effectiveness of an experimental drug called XH-S003 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that can cause fatigue, blood clots, and other serious problems. About 26 people who have already ta…

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

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 follows 64 Polish adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking ravulizumab as part of their routine care. Researchers will track blood markers like LDH and how many patients need blood transfusions over time. The goal is to see if the drug works…

This early-stage trial tests a new drug called VSA012 in about 50 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study includes people who have not used complement inhibitors before or who still have low hemoglobin …

This study tests a combination of two experimental drugs, pozelimab and cemdisiran, for people with PNH, a rare blood disease that destroys red blood cells. The goal is to see if this combo works better and is safer than current standard treatments (ravulizumab and eculizumab). A…

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 tests an experimental drug called LP-005 in 30 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The goal is to see if LP-005 can lower a key disease marker (LDH) and raise hemoglobin levels without needing bloo…

This study looks at the long-term safety of a drug called HRS-5965 for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It includes 132 patients who previously took the drug and benefited from it. Researchers will track side effects and measure how we…

This program offers continued access to a combination of two drugs, pozelimab and cemdisiran, for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare condition where red blood cells break apart. It is for patients who have already completed a related study and aims to m…

This Phase 2 trial tests NTQ5082, an oral drug, in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells break apart. The drug blocks a part of the immune system to prevent this destruction. The main goal is to see if it can raise h…

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that can cause dangerous blood clots. Researchers want to know how often PNH appears in people under 50 who have had a stroke, mini-stroke, or brain vein clot with no clear cause. The study will screen 200 partici…

This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease ou…