Hemophilia

This study compared two treatments to reduce bleeding after heart valve surgery: a concentrated clotting drug (PCC) versus fresh frozen plasma (FFP). The trial planned to enroll 476 adults undergoing elective valve surgery. It was terminated early, so the full comparison was not …

This early-stage trial tests a one-time treatment called BE-101 for adults with severe hemophilia B. The therapy uses a person's own blood cells, modified with CRISPR gene editing, to continuously produce the missing clotting factor. The goal is to reduce or prevent bleeding epis…

This study aimed to see if a new medicine (efanesoctocog alfa) helps people with moderate or severe hemophilia A reach their personal goals and be more physically active. It planned to enroll 35 people aged 12 to 50, but was stopped early after only 3 joined. The medicine is used…

This study tested two extra care programs—supportive palliative care and clinical management of other health issues—for older or frail patients who had a donor stem cell transplant. The goal was to see if these programs could improve quality of life and survival compared to stand…

This study looked at how patients with hemophilia A or those on blood thinners feel about monitoring their blood clotting at home. Researchers interviewed 19 people to understand their needs and hopes for a new device that could measure clotting factors. The goal was to gather in…