Hemophilia

This study is testing a new medicine called efanesoctocog alfa in about 90 people aged 12 and older with severe hemophilia A. Participants receive a weekly injection for 24 months to see if it helps them be more physically active and improves joint health. The study uses activity…

This study tested the safety of Jivi (BAY 94-9027), a longer-lasting clotting factor, in 36 children aged 7 to 12 with severe hemophilia A. The medicine is already approved for older patients, but this trial focused on younger kids to check for allergic reactions and whether the …

This study tested whether injecting bevacizumab (a drug that blocks blood vessel growth) into the joints of people with hemophilia can prevent repeated bleeding episodes. Eighteen participants with chronic synovitis (joint inflammation) received the injection. Researchers measure…

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 completed phase 2 trial tested a drug called foscenvivint in just 5 people with liver cirrhosis caused by HIV and hepatitis C co-infection who also have hemophilia. The goal was to see if the drug could improve liver function scores over 24 weeks. Because the study is very s…

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 emicizumab (Hemlibra) in 85 people with hemophilia A, a condition where blood doesn't clot properly, causing frequent bleeds. Participants received either emicizumab injections or no preventive treatment. The study found that those on emicizumab had sign…

This completed Phase 1 trial tested the safety of a new drug called Inno8 in 95 healthy men. Participants received either Inno8 or a placebo by injection or as tablets. The study measured side effects and how the drug moves through the body. Results will help decide if Inno8 shou…

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 two drug combinations in 110 adults with acquired hemophilia, a rare condition where the immune system attacks clotting factors, causing severe bleeding. One group received steroids plus cyclophosphamide, the other steroids plus rituximab. The goal was to see wh…

This study tested two treatments for acquired hemophilia A, a rare condition where the immune system attacks clotting factor VIII. 66 adults received either steroid plus a single dose of rituximab or steroid plus daily cyclophosphamide. The goal was to see which combination clear…

This study tested a medicine called emicizumab in 73 people with mild or moderate hemophilia A who do not have inhibitors. The goal was to see if it safely reduces the number of bleeding episodes. Participants received regular injections, and the results showed fewer bleeds compa…

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 medicine called turoctocog alfa pegol (N8-GP) in 36 Chinese men with severe hemophilia A, a bleeding disorder. The drug helps blood clot and was given as an injection to prevent or stop bleeding episodes. The trial lasted about 7-8 months and measured how many…

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 trial tested a long-acting clotting factor called N8-GP in 124 young children (under 6 years old) with severe hemophilia A who had never been treated before. The goal was to see if it safely prevents and treats bleeding episodes. The study found that N8-GP helped control ble…

This study tested a new medicine called Mim8 for people with hemophilia A, a condition where blood doesn't clot properly. Mim8 is given as a shot under the skin to help prevent bleeding episodes. The trial included 275 healthy volunteers and people with hemophilia A, with or with…

This study tested a new medicine called Mim8 in 281 adults and teens with hemophilia A, including those whose bodies make antibodies that block standard treatment. Mim8 helps replace the missing clotting factor and is given as a weekly or monthly injection under the skin. The goa…

This study looked at how safe it is for people with hemophilia A to switch from their current medicine (emicizumab) to a new one called Mim8. Mim8 helps prevent bleeding episodes by doing the job of the missing clotting factor. The study included 61 adults and teens who received …

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 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 tested a new scoring method to help doctors choose the best dosing schedule of Jivi (a longer-lasting clotting factor) for people with hemophilia A. 21 participants switched from standard treatments to Jivi, and their dose frequency was based on their personal risk sco…

This study tested a new medicine called Mim8, given as a shot under the skin, to prevent bleeding episodes in children aged 1-11 with hemophilia A (with or without inhibitors). The trial involved 70 participants and compared once-weekly and once-monthly dosing over about 54-98 we…

This study looked at how safe and effective Kovaltry is for people with hemophilia A when used in routine medical practice. Researchers tracked 230 patients for side effects and bleeding episodes. The goal was to gather real-world information, not to test a new treatment.

This study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The treatment aims to help the body produce its own clotting factor VIII, reducing the need for regular infusions. Participants also received steroids to manage immune re…

This study tested a new gene therapy (SB-525) in 13 adults with severe hemophilia A, a bleeding disorder. The goal was to see if a single infusion could safely raise the body's own clotting factor levels and reduce bleeding episodes. Participants were monitored for up to 5 years …

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 early study tested a new clotting factor called efanesoctocog alfa (BIVV001) in 13 adults with severe hemophilia A. The goal was to see how long it stays in the body compared to standard and extended half-life factors. Participants received single injections of each type in …

This phase 3 study tested a new medicine called BIVV001 (efanesoctocog alfa) in 159 people aged 12 and older with severe hemophilia A. The goal was to see if regular injections could prevent bleeding episodes, improve joint health, and enhance quality of life. Participants receiv…

This early-phase trial tested a gene therapy called BAX 888 in 4 men with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene for factor VIII, a blood-clotting protein they lack. Participants received a single intravenous infusion, and the study focus…

This study tested a new drug called BIVV001 in 74 children under 12 with severe hemophilia A, a condition where blood doesn't clot properly. The drug is given through a vein and aims to prevent bleeding episodes. The trial found that the drug was safe and effective at reducing bl…

This study looked at how the drug emicizumab (Hemlibra) works in real-world settings for infants and children up to 3 years old with hemophilia A, a bleeding disorder. Researchers tracked 71 children to see how many were using emicizumab to prevent bleeds and whether any breakthr…

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 tested whether a low-cost version of platelet-rich plasma (PRP) injections could safely ease joint pain and swelling in people with bleeding disorders like hemophilia. Fourteen adults with joint damage received the injection and were followed for 6 months. The goal was…

This study tested a physical therapy technique that uses a cuff to slightly restrict blood flow during exercise. The goal was to see if it could safely improve muscle strength and reduce pain in people with hemophilia who have ankle joint damage. 31 adults with hemophilia A or B …

This study tested a personalized program of exercise and manual therapy (like stretching and massage) in 10 adults with severe hemophilia who had multiple damaged joints. The goal was to see if it could reduce pain, improve joint movement and strength, boost heart-lung fitness, a…

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 tested a new drug called efanesoctocog alfa in 24 adults with severe hemophilia A. Researchers compared how long it stays in the body versus two existing drugs. Participants received one dose of their usual drug, then later one dose of the new drug, with blood samples …

This completed study observed how 329 Iraqi male patients with haemophilia A used turoctocog alfa (a clotting factor) in their daily care. Researchers reviewed medical records to see how the drug was given and how many bleeding episodes occurred. The goal was to describe real-wor…

This study tests a new medicine called Inno8 for hemophilia A. Researchers want to see how eating and drinking before and after taking the pill changes how much medicine gets into the blood. 80 healthy men aged 18-45 will take one dose after fasting, then wait different times bef…

This study watched 23 people with hemophilia A who were already using Esperoct as part of their normal care. Researchers tracked side effects and how well the medicine worked over about two years. The goal was to confirm the drug's safety and effectiveness in everyday use, not to…

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 observational study looked at boys aged 7-18 with moderate or severe hemophilia A in Canadian treatment centres. Researchers compared quality of life and caregiver burden between those receiving an intravenous factor replacement product and those on a subcutaneous …

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 study looked at how the COVID-19 lockdown changed the health habits and medical care of 1,343 people with chronic conditions like heart disease, multiple sclerosis, and hemophilia. Researchers used phone surveys to track medication use, lifestyle changes, and access to healt…

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…

This study tested whether a new pen injector delivers the drug Mim8 into the blood as well as a standard syringe and cartridge. 205 healthy men received a single injection of Mim8 using either the pen or the syringe. The goal was to see if the pen works just as well, making it ea…

This completed study from Hacettepe University compared lung function, exercise capacity, posture, and physical activity levels between 34 children with hemophilia and healthy controls. Researchers used spirometry, cycling tests, and joint health scores to see if hemophilia affec…