Inherited blood coagulation disorder

Atypical hemolytic uremic syndrome (aHUS) is a rare disease that damages the kidneys by causing blood clots in small vessels. The drug eculizumab blocks the complement system and has greatly reduced kidney failure, but doctors do not know how long patients should stay on it. This…

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.

Researchers are testing a long-acting factor VIII-Fc fusion protein called FRSW117 in adults and adolescents with severe hemophilia A. Participants receive the injection once a week for about a year, with extra doses as needed for bleeding. The trial measures how well this schedu…

This study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps preven…

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 tests whether aerobic exercise in simulated altitude (normobaric hypoxia) can improve body composition and metabolism in people with MASLD, a type of fatty liver disease. Twenty sedentary adults with MASLD will walk on a treadmill for 8 weeks, either in mild altitude o…

This trial is testing an investigational drug called PF-06741086, given as a once-weekly injection under the skin, to see if it can reduce the number of bleeding episodes in people with severe hemophilia A or moderately severe to severe hemophilia B. The study includes both peopl…

This study tests a gene therapy (PF-06838435) for people with severe hemophilia B, a bleeding disorder. The therapy uses a harmless virus to deliver a working gene for clotting factor IX. Researchers are following 21 participants long-term to see if it is safe and reduces bleedin…

This study tested a new oral medication called iptacopan in 34 adults with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots, low platelets, and kidney damage. The goal was to see if the drug could improve blood counts and kidney function w…

This trial compares two common methods for placing a totally implantable vascular access device (TIVAD), a port used for chemotherapy and other treatments in cancer patients. Researchers are randomly assigning patients to have the port inserted via either the internal jugular vei…

This phase 3 trial tests a long-acting clotting factor (rIX-FP) in 23 Chinese men with severe hemophilia B. The drug is given regularly to prevent bleeding episodes. Researchers will measure how well it works, how long it lasts in the body, and check for side effects like allergi…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This trial tests a gene therapy called valoctocogene roxaparvovec for people with severe hemophilia A who have developed inhibitors (antibodies that block standard treatment). The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor VIII, so the…

This study tests whether non-surgical gum treatment (deep cleaning) can improve oral health and quality of life in adults with hemophilia A or B. Researchers compare changes in gum health and daily comfort (using a questionnaire) between hemophilia patients and healthy individual…

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 lab study tested whether concizumab, a drug that blocks a natural clotting inhibitor, can improve blood clotting in people with Glanzmann thrombasthenia (GT), a rare genetic bleeding disorder. Blood samples from 20 adults with GT and healthy donors were mixed with concizumab…

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 tested a medicine called vonicog alfa (rVWF), sometimes given with another drug (ADVATE), to control bleeding in 47 children under 18 with severe von Willebrand disease. The treatment was given for 12-18 months, and doctors checked how well it stopped bleeding and if i…

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 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 study looked at how well the JETi device works in real-world settings to remove blood clots from deep leg veins (venous thrombosis). The device uses suction and tiny water jets to break up and remove clots. In 121 patients, the device successfully cleared at least 75% of the…

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 a medicine called wilate in 12 children under 6 years old with severe von Willebrand disease, a bleeding disorder. The goal was to see if regular infusions of wilate could prevent bleeding episodes over 12 months. Researchers measured how many bleeds occurred, h…

This study tested a medicine called BAX 930 in 52 people born with severe TTP, a rare condition that causes dangerous blood clots. The medicine replaces a missing enzyme to prevent or treat sudden flare-ups. Participants received either BAX 930 or standard treatment for 6 months,…

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 tested the drug eculizumab in 25 children and adults in China with a rare kidney condition called atypical hemolytic uremic syndrome (aHUS). The goal was to see if the drug could stop blood clots and improve kidney function. All participants received eculizumab intrave…

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 whether taking a protein supplement on fasting days during a 4-week alternate-day fasting program could help young men with overweight lose fat while keeping muscle. Twenty-six men aged 21 to 35 either ate a small meal (400-600 kcal) or the same meal plus a w…

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 completed phase 3 trial tested whether adding tranexamic acid to recombinant von Willebrand factor (rVWF) reduces postpartum hemorrhage better than rVWF alone in 20 pregnant women with von Willebrand disease. Participants received either the combination or rVWF alone at deli…

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-stage trial tested a single injection of a new drug called BIVV001 in 6 adults with severe forms of von Willebrand disease (types 2N and 3). The goal was to see how the drug behaves in the body and whether it is safe. If results are promising, larger studies could foll…

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 tested a drug called rVWF (Vonvendi) in 38 children and adults with severe von Willebrand disease, a bleeding disorder. The goal was to see if regular use of the drug could reduce the number of spontaneous bleeding episodes over a year. Participants received treatment …

This study tested a new lab method to diagnose platelet function disorders in 322 people with bleeding symptoms. The method uses flow cytometry to detect signaling defects in platelets, which are often missed by standard tests. The goal was to standardize the technique across mul…

This trial tests whether a short 5-day course of the blood thinners rivaroxaban (Xarelto) or apixaban (Eliquis) can prevent blood clots that sometimes form after laser treatment for varicose veins. Adults with symptomatic vein reflux undergoing laser ablation of the great sapheno…

This study compared three common blood thinners—aspirin, warfarin, and rivaroxaban—to prevent dangerous blood clots after hip or knee replacement surgery. Nearly 19,000 adults took part. The goal was to find which option best balances preventing clots with avoiding bleeding side …

This study looked at 86 women with Factor V Leiden or Factor II mutations who had no history of blood clots. Researchers compared how long doctors prescribed blood thinners after childbirth (less than 2 weeks vs. 6 weeks) to see if shorter treatment was still effective at prevent…

Boys with hemophilia often miss out on games and activities because of concerns about bleeding and joint damage. Researchers developed a 12-week muscle-strengthening exercise programme with input from children, parents, and physiotherapists. The DOLPHIN-II trial compares this pro…

This study tests whether a single injection of nanofat—made from a person's own fat—can improve the appearance of atrophic scars caused by burns or trauma. Each participant has two similar scars: one gets the nanofat injection plus silicone gel, and the other gets only silicone g…

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…

Researchers at Nantes University Hospital are studying how joint damage from bleeding affects walking in adults with moderate hemophilia. About 24 participants will have their gait measured with motion capture and force platforms during a single visit. The study also collects cli…

This study reviews medical records of 200 people diagnosed with a rare blood and kidney disease called aHUS in Egyptian hospitals. Researchers want to learn about patient symptoms, treatments, and long-term health to help doctors diagnose and manage the disease better. No new tre…

This study aims to understand how ultra-processed foods affect energy metabolism compared to unprocessed foods. Healthy adults aged 18-60 will stay at a research center for four weeks, eating different diets each week. Researchers will measure how much they eat, their metabolic r…

This observational study will look at 138 adults with hemophilia and joint damage to understand how central pain mechanisms and psychological factors like anxiety affect pain interference in daily activities. Researchers will measure pain intensity, sleep quality, and pain self-e…

This study follows people with severe von Willebrand disease (VWD) to see how well different treatments work and how safe they are in everyday use. Researchers are tracking side effects and how the body responds to therapy given on demand, during surgery, or as regular prevention…

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 examined blood samples from 119 adults with sickle cell disease, sickle cell trait, or no condition to understand why some develop dangerous blood clots. Participants gave blood and had health check-ins over two years. The goal was to find biomarkers that could lead to…

This study examined the DNA of 254 people to find genetic changes that might explain why some individuals have mildly low levels of von Willebrand factor, a protein important for blood clotting. Researchers used whole-exome sequencing to look for variants in known and new genes. …

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 28 people with Glanzmann thrombasthenia, a rare bleeding disorder, to understand why some develop harmful antibodies after blood transfusions or pregnancy. Researchers checked for antibodies every six months and after each transfusion over 18 months. The goal…

This study looked at whether using a video capsule endoscopy (a small camera pill) along with standard scopes can better find the source of gut bleeding in people with von Willebrand disease. Researchers reviewed records of 50 patients who had gut bleeding between 2015 and 2017. …

This study examined how the drug tranexamic acid (TXA) affects blood clotting in women having planned C-sections. Researchers measured clot breakdown times in 34 participants to see if TXA could help prevent heavy bleeding after delivery. The goal was to better understand how TXA…

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 looked at whether intimate partner violence (IPV) increases the risk of blood clots in women taking combined oral contraceptives. Researchers compared 997 women who had a first blood clot with similar women who did not, using a questionnaire to screen for IPV…

This study looked at 257 emergency patients on ECMO (a heart-lung machine) to understand why many experience serious bleeding. Researchers measured how often bleeding occurred and checked for defects in von Willebrand factor, a key clotting protein. The goal is to find links betw…

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 looked at how common major psychiatric disorders are in women with antiphospholipid syndrome (a blood clotting disorder) and similar pregnancy loss histories. Researchers compared three groups of women about 10 years after their initial diagnosis. The goal was to under…

This study measured the amount of free-floating DNA and nucleosomes in the blood of 137 women to see if these levels differ between those with pregnancy complications (like preeclampsia or blood clots) and healthy women (both pregnant and non-pregnant). The goal was to better und…

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 aimed to understand the real-life experiences of people with von Willebrand disease, a common but often overlooked bleeding disorder. Over 600 adults from the UK, Ireland, and the US shared their stories through online surveys, one-on-one interviews, and a 30…

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…