Paroxysmal nocturnal hemoglobinuria

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This study tracks 8 Korean patients with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Danicopan alongside standard C5 inhibitors like eculizumab or ravulizumab. The goal is to monitor safety and check if the drug works as expected in everyday medical practice. It is a…

This study tests a medicine called iptacopan in children aged 2 to under 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. About 12 children will take the medicine, and doctors will monitor side effects and blood…

This study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose red blood cell destruction is not well-controlled by current C5 inhibitors. The goal is to see if the combination can lower hemolysis (bre…

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive eit…

This study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the…

This study is monitoring the safety and effectiveness of the drug iptacopan (Fabhalta) in 21 Korean adults with paroxysmal nocturnal hemoglobinuria (PNH) or C3 glomerulopathy (C3G). Researchers will track side effects and lab results from routine medical records. The goal is to s…

This study tests a new drug called ADX-038 in healthy volunteers and people with PNH who still have low red blood cells despite treatment. The goal is to check safety and how the drug works in the body. It is an early-stage trial with 50 participants.

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, an…

This phase 2 trial tests a bone marrow transplant using lower doses of chemotherapy and radiation to prepare the body, along with a drug called cyclophosphamide after transplant to prevent graft-versus-host disease. It enrolls 60 people with severe aplastic anemia or related bone…

This study looks at how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track changes in hemoglobin levels and other health outcomes in 80 patients across China. The goal is to see if iptacopan impro…

This early-stage trial tests the safety of a new drug called EA5 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug is an antibody designed to block part of the immune system that attacks blood cells. The goa…

This study tests a new drug called HS-10542 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The goal is to see if the drug can safely raise hemoglobin levels and reduce the need for blood transfusions. About 50 partic…

This phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participa…

This program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exha…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

This study uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

This early-stage trial is testing an experimental oral drug called TUL321 in healthy adults to see if it is safe and tolerable. The study will also measure how the drug moves through the body and whether food affects its absorption. TUL321 is being developed as a potential treatm…

This study looks at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The go…

This study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This early-stage study tests a new drug called CMS-D017 in 88 healthy adults to see if it is safe and how the body processes it. The drug is being developed for two rare conditions: paroxysmal nocturnal hemoglobinuria (a blood disorder) and complement-mediated kidney disease. Par…

This study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…

This early-stage study is testing a single dose of the drug HSK39297 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH) who have mild or moderate liver impairment, compared to those with normal liver function. The goal is to see how the liver affects drug levels and safe…