HEMOPHILIA A

This observational study tracks 33 adults with hemophilia A who choose to take Jivi, a longer-lasting clotting factor. Researchers use ultrasound to see if joint health improves or stays stable after 24 months of treatment. The goal is to understand how well Jivi works in everyda…

This study follows about 200 people with hemophilia A who are taking a new medication called efanesoctocog alfa as part of their regular care. Researchers want to see how well it protects joints from bleeding over 5 years. No extra treatments are given—just observation of routine…

This early-stage 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 a safe dose that raises clotting factor levels …

This study looks at the long-term safety of the medicine ADYNOVI/ADYNOVATE in people with hemophilia A. It involves 207 participants who already use this drug to prevent bleeding. The main goal is to track any side effects, especially those that might affect the kidneys, liver, o…

This early-phase study tests a gene therapy for severe hemophilia A in 5 adult men with a history of inhibitors. Doctors take the patient's own blood stem cells, add a gene that makes factor VIII, and return the modified cells. The goal is to see if this approach is safe and can …

This study follows people with severe hemophilia A who received a one-time gene therapy (BMN 270) in earlier trials. Researchers want to see how safe the treatment is over many years and whether it continues to reduce bleeding episodes. About 172 participants will be monitored be…

This study follows 427 people with hemophilia A who are already taking Elocta to prevent bleeding. Over 48 months, researchers will track joint health, including bleeding episodes and joint damage. The goal is to see how well Elocta works in everyday life to protect joints.

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 aimed to compare two standard treatments for severe hemophilia A—extended half-life factor VIII and non-factor VIII therapies—to see which better protects joint health and prevents bleeding. It planned to enroll children under 18 without existing joint damage. However,…

This study follows 62 people with hemophilia A who are already using Jivi (damoctocog alfa pegol) to prevent bleeding. Researchers will collect safety data during routine doctor visits for at least 4 years. The goal is to better understand long-term side effects of this extended …

This study looks at whether checking the amount of emicizumab in the blood can help predict the risk of bleeding in people with severe hemophilia A. Researchers will follow 100 adults and children who are already taking emicizumab and track any bleeding events over about a year. …

This study watches how Jivi (damoctocog alfa pegol) is used in routine care for people with hemophilia A. It tracks how many bleeds occur, any side effects, and how satisfied patients and doctors are with the treatment. About 371 people who have used Jivi before are taking part, …