PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

This program provides access to a combination of two drugs, pozelimab and cemdisiran, for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare immune disease that destroys red blood cells. It is for patients who have already completed a related study and aims to monitor …

This study tests a new drug called crovalimab against the current standard treatment eculizumab in about 190 people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Participants must already be on complement inhibitor therapy. The goal is to see if crovalima…

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. Participants are those who already took HRS-5965 in an earlier study and benefited from it. They will continue taking the drug to s…

This study tests whether a new drug called crovalimab works as well as the current standard treatment eculizumab for people with PNH, a rare blood disease that destroys red blood cells. About 210 adults who have not received prior complement inhibitor therapy will be randomly ass…

This study tests a combination of two experimental drugs, pozelimab and cemdisiran, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The goal is to see if this new combo works better and is safer than current standard treatments (ravulizumab and ec…

This study tests a new medicine called crovalimab in about 50 people with a rare blood disease called PNH. The goal is to see if the drug can control the breakdown of red blood cells and reduce the need for blood transfusions. Participants must be at least 12 years old and have n…

This study tests an experimental capsule called NTQ5082 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease where red blood cells break apart. The drug blocks a part of the immune system to stop red blood cell destruction. The goal is to see if it sa…

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 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 looks at the long-term safety of iptacopan in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 208 adults who completed earlier iptacopan trials will continue taking the drug and be monitored for side effects and how well it controls …

This study watches 132 people with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking Fabhalta. Researchers track infections, side effects, and what happens if treatment stops. No new drug is tested—the goal is to learn more about safety in everyday use.