Hemophilia B

This study follows 9 adults with severe or moderately severe hemophilia B who received a one-time gene therapy (AAV5-hFIX) to help their bodies produce clotting factor IX. Researchers are checking if the treatment remains safe and effective for up to 10 years after the initial do…

This study followed 28 people with hemophilia B in Japan who were already prescribed Refixia as part of their normal care. Researchers tracked side effects, bleeding episodes, and quality of life to confirm the drug's safety and effectiveness over time. The study did not test a n…

This study tested a freeze-dried plasma product that can be mixed with water in 6 minutes, avoiding the long thaw time of standard plasma. It included 42 trauma patients with severe bleeding who needed blood transfusions. The goal was to see if faster plasma delivery could improv…

This study looked at whether giving the antibiotic vancomycin helps cancer patients who have diarrhea and a positive screening test for C. difficile but a negative toxin test. Only 9 patients were enrolled, far fewer than planned. Researchers measured changes in C. difficile leve…

This early-stage study tested whether adding a drug called BMS-986004 to standard immune-suppressing medicine could help prevent acute graft-versus-host disease (GVHD) in 45 adults with blood cancers or disorders receiving a stem cell transplant. GVHD occurs when donor immune cel…

This study tested a new gene therapy for adults with severe hemophilia B, a bleeding disorder caused by a faulty gene. The treatment uses a harmless virus to deliver a working copy of the factor IX gene, aiming to reduce bleeding episodes. Fourteen participants received one of fo…

This phase 3 trial tested a one-time gene therapy called AMT-061 in 67 men with severe or moderately severe hemophilia B. The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor IX, so the body can produce its own. The main goal was to see if i…

This study tested whether a hydrochloric acid lock can help treat central line infections in 212 cancer and blood disease patients. The acid solution was placed in the line for 10 minutes, repeated three times, alongside standard antibiotics. The goal was to see if it reduces tre…

This study tested a medicine called nonacog beta pegol (N9-GP) in 30 Chinese men with moderate to severe hemophilia B. The medicine is injected into a vein to help blood clot and prevent or treat bleeding episodes. The trial lasted about 12-16 months and checked how well the medi…

This study tested a new long-acting factor IX drug (nonacog beta pegol) in 25 children with hemophilia B. The drug is given as a weekly injection to prevent bleeding episodes. The main goal was to check safety and whether the body develops antibodies that block the treatment. Res…

This study tested a new long-acting clotting factor drug called nonacog beta pegol in 54 young children with hemophilia B who had not been treated before. The drug was given regularly to prevent bleeding episodes and also to treat any bleeds that occurred. The main goal was to se…

This early-phase study tested a new drug called MG1113, given as a shot under the skin, in 15 adults with severe hemophilia A or B. The main goal was to check safety and how the drug moves through the body. Participants received multiple doses to see if the drug is well-tolerated…

This study tested a pathogen-reduced version of cryoprecipitate, a blood product that helps blood clot, in 208 patients undergoing liver transplant or heart surgery. The goal was to see if having this product readily available could stop bleeding early and reduce the need for oth…

This study looked at 50 patients who needed a stem cell transplant from a partially matched donor but had antibodies that could attack the donor cells. Before transplant, they received a blood purification treatment called protein A immunoadsorption to remove those antibodies, so…

This study followed 151 people with hemophilia B who were already using or starting Alprolix, a long-lasting clotting factor. Over 24 months, researchers tracked how often bleeding occurred, how often the drug was injected, and how much was used. The goal was to see how well Alpr…

This study looked at whether touch-massage can reduce anxiety in people with blood disorders (like leukemia or lymphoma) who are hospitalized in a sterile, isolated room. Sixty-two patients received three massage sessions, and researchers measured their anxiety levels before and …

This study tested whether a 6-month adapted physical activity program could improve functional capacity in people with hemophilia. Fourteen participants did exercises twice a week to improve joint mobility, strength, and balance. The main goal was to see if their walking distance…

This study looked at whether a special low-intensity exercise program, using blood flow restriction cuffs, is safe and helpful for people with moderate or severe hemophilia. Twenty adults completed 8 weeks of arm and leg exercises. Researchers measured changes in muscle function,…

This study looked at whether manual therapy (hands-on treatment) and structured exercises could improve balance and walking ability in adults with hemophilia. Thirty-five adults with moderate or severe hemophilia took part. Half received 12 physiotherapy sessions over 6 weeks, wh…

This study tested a monthly online peer support group for young adults (ages 18-39) who had received a stem cell transplant. The group met for one hour over six months, led by a nurse and social worker. The goal was to see if the program was practical and helpful for reducing anx…

This study tested whether montelukast (Singulair), a common asthma drug, can reduce the number and severity of infusion reactions in people receiving monoclonal antibody treatments for blood disorders. Forty adults were given montelukast along with standard premedication before t…

This study looked for a virus called HHV6 in the skin and blood of 37 adults with blood cancers who developed a rash during hospital stays. The goal was to see if the virus is linked to the rash, which could help doctors diagnose and treat the rash more effectively. The study is …

This study looked at bone density in 448 men with hemophilia and healthy volunteers to see how common bone loss is. Participants had a bone density scan and some gave blood samples. The goal was to understand if hemophilia itself, not just lack of activity, leads to weaker bones.

This study looked at whether it is safe to lower the number of platelets needed before placing a central line (a special IV) in patients with blood disorders. Normally, patients get a platelet transfusion if their count is below 50, but this study tested a lower trigger of 10. Th…

This completed study involved 15 people with severe haemophilia B who were already taking eftrenonacog-alfa, a clotting factor medicine. Researchers compared different lab tests to see which ones best measure factor IX activity in real-life conditions. The goal was to improve mon…

This study looked at why people with hemophilia and damaged knee or ankle joints feel ongoing pain. Researchers measured pain sensitivity, fear of movement, and anxiety in 146 adults. The goal was to better understand central sensitization—a condition where the nervous system sta…

This completed study tested whether artificial intelligence and MRI scans could help automate the planning of total marrow irradiation, a type of radiation therapy given before a bone marrow transplant for blood cancers. Researchers aimed to reduce the 10-day planning time and be…

This study looked at over 1,200 people in Denmark who had a stem cell transplant for blood diseases, some as far back as 1970. Researchers used detailed surveys to measure late effects and quality of life. The goal is to find out what medical and personal factors help survivors f…

This completed study examined lung CT scans from 148 adults with blood diseases to identify common patterns of lung involvement. Researchers tracked how often certain radiological features appeared and counted complications. The goal was to better understand the range of lung iss…

This study measured static and dynamic balance in 41 adults with hemophilia and 40 healthy volunteers using a special platform. Researchers also looked at how joint health, physical activity, and fear of movement affect balance. The goal is to understand why balance may be worse …

This study interviewed 150 people with hemophilia B, their caregivers, and healthcare workers from around the world to learn what they know and how they feel about gene therapy. The goal is to create better educational materials and improve the consent process, especially in coun…

This study gathered information on how often adults with moderate to severe hemophilia A or B experience bleeding while on their usual treatment. The data will be used as a baseline for future gene therapy studies. About 212 participants took part, and no experimental treatment w…