Severe hemophilia A

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 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 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 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 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 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 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 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 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 …