HEMOPHILIA B

This early-stage study is testing a new, one-time gene therapy called BE-101 for adults with moderately severe or severe hemophilia B. The goal is to see if a single infusion of a patient's own genetically edited cells can safely help the body produce its own missing clotting fac…

This study is testing a one-time experimental gene therapy called REGV131-LNP1265 for people with hemophilia B, a genetic bleeding disorder. The therapy uses CRISPR technology to insert a working gene into a person's DNA, aiming to teach their body to produce its own blood clotti…

This study is testing a one-time gene therapy called CSL222 in adults with severe hemophilia B who have pre-existing antibodies that normally block this type of treatment. The main goal is to see if the therapy safely reduces bleeding episodes and the need for regular clotting fa…

This study aims to evaluate the long-term safety and effectiveness of marstacimab, a preventive injection treatment, for people with severe hemophilia A or B. It includes about 245 participants, both children and adults, who have already been in previous marstacimab studies. The …

This study is testing a single-dose gene therapy called CSL222 in teenage boys with severe hemophilia B. The goal is to see if this one-time treatment can help their bodies produce their own clotting factor, reducing or eliminating the need for regular infusions to prevent bleeds…

This study is testing whether a single dose of an experimental gene therapy called ANB-002 can control bleeding as well as, or better than, the standard preventive injections of clotting factor (FIX) in adults with severe hemophilia B. Participants will first be monitored for 6 m…

This is the first human study testing a new drug called SR604. Researchers want to see if it's safe and how it behaves in the body. First, healthy volunteers will receive single doses. Then, people with hemophilia A, hemophilia B, or Factor VII deficiency will receive multiple do…

This study follows 500 people with hemophilia B who have received HEMGENIX gene therapy to understand its long-term effectiveness and safety. Researchers will track how many bleeding episodes patients experience and compare results to those receiving standard preventive treatment…

This study is evaluating a new, non-invasive blood test to diagnose serious inherited genetic disorders in unborn babies. The test analyzes tiny bits of the baby's DNA found in the mother's blood, aiming to provide a safe and accurate diagnosis as early as 9 weeks into pregnancy.…

This study aims to gather detailed information on many different blood diseases in China, including cancers like leukemia and lymphoma, and conditions like hemophilia. It will follow 2,300 patients to learn how common these diseases are, what treatments patients receive, how well…

A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data colle…