Acquired metabolic disease

This early-phase trial tested a combination of three drugs—daratumumab, ixazomib, and dexamethasone—in 21 people with AL amyloidosis, a rare disease where abnormal protein builds up in organs. The main goal was to find the safest dose and check for side effects. Researchers also …

This completed Phase 3 trial tested ravulizumab in 18 adults in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study measured changes in LDH, a marker of red blood cell breakdown, to see if the drug could control the…

This study tested the long-term safety of a drug called pegcetacoplan in people with PNH, a rare blood disease that destroys red blood cells. All 137 participants had already completed a prior pegcetacoplan study and continued treatment. The main goal was to track side effects ov…

This study tested eculizumab in 25 adults with PNH in China who had not previously taken complement inhibitors. Participants received infusions over 62 weeks. The main goal was to see if the drug reduced LDH levels, a marker of disease activity. Results showed a decrease in LDH, …

This early study tested an experimental drug called OMS906 (zaltenibart) in 15 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease where red blood cells break apart. The main goal was to check safety and how the body handles the drug. Researchers also look…

This study followed 120 adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from the older drug eculizumab to the newer ravulizumab. Researchers measured changes in a blood marker called LDH to see if the new drug controlled red blood cell breakdown just as well or…

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This study tested a new tablet called HSK39297 in 36 adults with paroxysmal nocturnal hemoglobinuria (PNH) who still had anemia despite stable treatment with anti-C5 antibodies. The goal was to see if the pill could raise hemoglobin levels and reduce the need for blood transfusio…

This study tested an oral medication called iptacopan in 52 adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who were already stable on standard infusion treatments. Participants switched from their usual therapy to iptacopan pills taken twice daily. The main goal was to see…

This phase 3 trial tested a new drug called HRS-5965 in 39 adults with paroxysmal nocturnal hemoglobinuria (PNH) who remained anemic even after standard anti-C5 antibody treatment. The study aimed to see if the drug could raise hemoglobin levels without needing blood transfusions…

This study tested a new pill called HSK39297 against a standard infusion drug (eculizumab) in 73 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. Participants had not received prior complement inhibitor treatment. The mai…

This study tested whether adding daratumumab to standard chemotherapy (CyBorD) helps people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. About 416 adults took part. The goal was to see if the combination leads to better blood res…

This study tested a new drug called OMS906 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who were not getting enough benefit from the standard treatment ravulizumab. The goal was to see if OMS906 is safe and can improve hemoglobin levels. Participants received OMS90…

This Phase 3 study tested a new oral medication, HRS-5965, against the standard infusion eculizumab in 76 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The main goal was to see if HRS-5965 could raise hemoglobin levels to normal without needing blo…

This phase II trial tested the drug isatuximab in 43 patients with AL amyloidosis that had come back or stopped responding to treatment. Isatuximab is a monoclonal antibody that may help stop abnormal protein buildup. The study measured how well the drug reduced disease markers a…

This phase 2 study tested a drug called belantamab mafodotin in 35 people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Participants had already tried other treatments without success. The goal was to see if the drug could reduce or eliminate tho…

This completed Phase 3 study tested whether a radioactive tracer called [18F]Florbetaben, used in PET scans, can accurately diagnose cardiac AL amyloidosis—a condition where abnormal proteins build up in the heart. 244 adults with suspected cardiac amyloidosis received a single P…

This pilot study tested whether a radioactive tracer called F-18 florbetapir (Amyvid) can detect amyloid protein buildup in the heart using a PET scan. Researchers enrolled 23 people with known cardiac amyloidosis and a control group without the condition. The goal was to see if …

This phase 2 trial tested a radioactive tracer called 124I-AT-01 to see how well it can measure amyloid buildup in organs like the heart, kidneys, liver, and spleen using PET/CT scans. 33 adults with systemic amyloidosis received the tracer twice, six weeks apart. The goal was to…

This study uses advanced heart imaging to understand how blood flows inside the heart in people with cardiac amyloidosis, a condition where abnormal proteins build up in heart tissue. Researchers will compare these images with those from healthy volunteers to find unique patterns…

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This study tested whether a special MRI scan of the wrist can detect amyloid deposits in people with systemic amyloidosis, a condition where abnormal proteins build up in organs. Researchers enrolled 128 adults, including patients with cardiac amyloidosis and healthy volunteers. …

This completed study involved 250 adults with AL amyloidosis, a rare disease where abnormal proteins damage organs. Researchers analyzed patient samples and data to find biological markers that predict how well someone will respond to initial treatments. The goal is to help docto…

This study looked at 30 people newly diagnosed with cardiac AL amyloidosis, a rare disease where abnormal proteins damage the heart. Researchers used advanced MRI, echocardiogram, and PET scans to see if these images could predict survival and show how well chemotherapy works. Th…

This study tested whether a special PET imaging probe called [68Ga]CBP8 can measure scarring (fibrosis) in the hearts of people with cardiac amyloidosis, a rare condition where abnormal proteins build up in the heart. Researchers compared 18 participants with cardiac amyloidosis …

This study followed 37 people with a genetic risk for hereditary ATTR amyloidosis over five years. Researchers measured levels of misfolded proteins in the blood to see if they could detect the earliest signs of the disease. The goal is to develop a way to catch the condition bef…

This study collected health information from over 6,000 people with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The goal was to track the safety of two medications, Soliris and Ultomiris, and to understand how PNH changes over time. Participants were followe…

This completed study looked at 110 people with cardiac amyloidosis, a condition where protein deposits build up in the heart. Researchers implanted a small device under the skin to continuously monitor heart rhythms for up to several years. The goal was to learn more about when a…

This completed study followed 724 people in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers aimed to better understand how the disease progresses and how patients respond to different treatments, including the newly available drug eculizum…

This early-stage study tested a new drug, LP-005, in 68 healthy adults aged 18 to 50. The main goal was to check if the drug is safe and how the body handles it. This information will help design future studies for people with a rare blood disorder called PNH.

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…