Inherited blood coagulation disorder

This trial tests a gene therapy called FLT180a in adults with hemophilia B, a condition where the body lacks a clotting protein. The therapy delivers a working gene to help the body produce its own clotting factor. A small group of participants will receive a single dose and be f…

This trial tests whether a medication called eptacog beta can safely prevent excessive bleeding in people with hemophilia A or B who have inhibitors and need major surgery. Participants receive the drug during and after their operation, and doctors measure how well bleeding is co…

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 a drug called narsoplimab (OMS721) for atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and organ damage. The trial aimed to see if the drug could improve platelet counts and was safe for adults and adolescents. However, the st…

This study tested a combination of two drugs, atezolizumab and bevacizumab, in 46 people with advanced colorectal cancer that had stopped responding to chemotherapy and had a specific genetic feature called MSI-like. The goal was to see if the treatment could shrink tumors. The s…

This study checks on people who previously received gene editing for hemophilia B or mucopolysaccharidosis (MPS) I or II. No new treatment is given; instead, participants are monitored for up to 10 years to see if any new health problems or worsening of existing conditions appear…

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…