HEMOPHILIA A

This study looks at adults with hemophilia A who choose to take Jivi, a longer-lasting clotting factor, to prevent bleeding. Researchers will use ultrasound to check joint health after 24 months of treatment. The goal is to see if Jivi helps maintain or improve joint status in ev…

This study tests a gene therapy called BAY2599023 in 11 adults with severe hemophilia A. The treatment delivers a healthy copy of the factor VIII gene so the body can produce its own clotting protein. The goal is to find the safest and most effective dose to reduce or stop bleedi…

This early-phase study tests a new gene therapy for adults with severe hemophilia A who have or had inhibitors. Doctors take the patient's own stem cells, add a gene that makes Factor VIII (a clotting protein), and return the cells. The goal is to see if this is safe and if the m…

This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to see if it can raise their clotting factor levels and reduce or stop the need for regular factor VIII infusions. Participants receive the therapy via IV…

This study follows about 200 people with hemophilia A who are already using efanesoctocog alfa as part of their routine care. Researchers will track joint bleeding and joint health over up to 5 years to see how well the treatment works in real life. No experimental drugs are give…

This 48-month observational study looks at how well Elocta, a preventive treatment for hemophilia A, protects joints over time. About 427 people with hemophilia A who already use Elocta will be followed to track joint bleeds, target joint issues, and joint damage. The goal is to …

This study monitors 207 people with hemophilia A who are taking ADYNOVI/ADYNOVATE as part of their regular care. The goal is to track any long-term side effects, including problems with kidneys, liver, or nerves. It does not test if the drug works, but rather how safe it is over …

This study follows 172 people with severe hemophilia A who received a one-time gene therapy (BMN 270) in an earlier trial. Researchers want to see if the treatment stays safe and effective beyond 5 years. Participants may be on other hemophilia treatments and still join.

This study looks at how the drug Jivi (damoctocog alfa pegol) works for people with Hemophilia A in real-world settings, not just in controlled trials. Researchers are tracking bleeding episodes, side effects, and how satisfied patients and doctors are with the treatment. About 3…

This study was designed to compare two standard treatments for severe or moderate hemophilia A: extended half-life factor VIII therapy and non-factor VIII therapy. Researchers planned to use ultrasound and blood tests to see which treatment better protects joints and prevents ble…

This study watches people with hemophilia A who are already using Jivi (a long-acting clotting factor) to prevent bleeding. Researchers will collect safety information during regular doctor visits for at least 4 years. No new treatment is given—just observation of real-world use.…