Acquired metabolic disease

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 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…

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 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 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 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 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 Phase 3 trial tests whether a daily injection of metreleptin (a lab-made leptin hormone) can improve blood sugar and fat levels in people with partial lipodystrophy, a rare condition where fat is distributed abnormally. About 69 participants will receive either metreleptin o…

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 combination of three drugs—venetoclax, ixazomib, and dexamethasone—in 24 patients with light chain amyloidosis that has returned or not responded to prior treatment. The study focuses on finding the safest dose and checking side effects. All partici…

This phase 2 trial tests a drug combination including daratumumab for people with AL amyloidosis, a rare disease where abnormal proteins damage organs, especially the heart. The study has two groups: one gets the full combo right away, the other starts some drugs later. Researche…

This study tests a new medicine, crovalimab, for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that destroys red blood cells. It involves healthy volunteers and people with PNH to check safety and how well the drug works. The goal is to control the disease, not …

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 is for people with systemic amyloidosis, a disease where abnormal proteins build up in organs. It tests the long-term safety of an experimental drug called AT-02. About 120 adults will receive the drug and be monitored for side effects and how the drug behaves in the b…

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 whether semaglutide, a drug used for diabetes, can reduce harmful belly fat and improve heart health in people with HIV who have excess fat around the midsection (lipohypertrophy). About 108 participants will receive either semaglutide or a placebo for 32 weeks. …

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 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 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 trial aimed to test an experimental drug called CM-336 for people with AL amyloidosis that has returned, not responded to treatment, or only partially responded after initial therapy. The drug works by helping the immune system attack and destroy harmful cells. However, the …

This study tests whether fecal microbiota transplant (FMT) capsules are safe and doable for people with auto-brewery syndrome, a rare condition where gut microbes turn carbs into alcohol. Eight adults will take antibiotics and a colon cleanse, then receive five FMT capsules over …

This study tests whether cyclophosphamide, a drug that calms the immune system, can reduce insulin needs in people with a rare condition that causes fat loss (lipodystrophy) and type 1 diabetes. Ten participants will receive the treatment, and researchers will measure how much in…

This study tests whether adding daratumumab back to pomalidomide and dexamethasone can improve complete remission rates in 15 adults with relapsed or refractory AL amyloidosis who have already received daratumumab. Participants receive the three-drug combination to control the di…

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 early-phase trial tests whether adding the drug isatuximab to standard chemotherapy can safely treat people with high-risk AL amyloidosis, a rare disease where abnormal proteins damage organs. Eleven participants will receive the combination to see if it reduces toxicity and…

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 a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

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 phase 2 trial is testing whether the oral chemotherapy drug ixazomib can help control AL amyloidosis after initial treatment. Seventeen adults with the disease will take ixazomib along with low-dose dexamethasone to see if it delays organ progression or the need for more the…

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 study tests a new, more stable version of the chemotherapy drug melphalan in people with multiple myeloma or AL amyloidosis who are getting a stem cell transplant. The goal is to find the best dose that reaches a target level in the body, while possibly causing fewer side ef…

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…

This study tests whether a drug called siltuximab can reduce symptoms like weakness, fatigue, nausea, and pain after a stem cell transplant in patients aged 60-75 with multiple myeloma or AL amyloidosis. The drug blocks a protein linked to inflammation, which may help patients re…

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…