Severe hemophilia A

This Phase 3 trial tests a new drug called NXT007 (Zemocimig) against standard Factor VIII treatment to prevent bleeding in people with severe or moderate hemophilia A who do not have inhibitors. About 126 participants aged 12 and older will receive either NXT007 as a shot under …

This phase 3 trial is testing a drug called fitusiran in about 85 boys aged 1 to under 12 with severe hemophilia A or B, including those with inhibitors that make standard treatments less effective. The drug is given as a regular shot under the skin to help prevent bleeding episo…

This study tests a new medicine called marstacimab in children aged 1 to 17 with severe hemophilia A or B. The medicine is given as a weekly shot under the skin to prevent bleeding episodes. Researchers will compare how well it works to the children's own past year of standard tr…

This study looks at the long-term safety and effectiveness of a medicine called marstacimab for people with severe hemophilia A or B. Participants receive regular injections to help prevent bleeding episodes. The goal is to see if this treatment is safe and reduces bleeds over ti…

This study gives people with moderate or severe hemophilia A who finished a previous trial continued access to the drug efanesoctocog alfa. The drug is a lab-made clotting factor that helps prevent bleeding episodes. Researchers will monitor safety and how well it controls bleedi…

This study looks at whether it is safe for males aged 12 and older with severe hemophilia A to switch from their current medicine (emicizumab) to a new one called fitusiran. About 20 participants will be followed for up to 28 months to check for side effects and how well the new …

This early-stage study tests a new medicine called marstacimab in people with severe hemophilia A who want to switch from their current preventive treatment, emicizumab. The study includes 15 adolescents and adults who will receive weekly injections of marstacimab for 4 months. R…

This study tests whether a recombinant factor VIII product called Nuwiq can safely prevent bleeding during major surgery in people with severe hemophilia A who are already on emicizumab. About 28 male patients aged 12 and older will take part. The main goal is to see if the treat…

This study is testing a drug called SCT800, a lab-made clotting factor, to prevent bleeding episodes in 36 boys with severe hemophilia A who have never been treated before. Participants receive regular infusions of SCT800, and researchers will monitor how many bleeding events occ…

This study is observing how well three Factor VIII concentrates (Nuwiq, Octanate, Wilate) work in real-world settings for people with severe hemophilia A who have had little or no prior treatment. Researchers will track bleeding rates and side effects in about 200 participants of…

This study aims to create a reliable lab test to monitor how well anti-TFPI antibodies work in people with severe hemophilia. Researchers will take a single blood sample from 11 patients already on anti-TFPI therapy and test it in the lab. The goal is to better predict and manage…

This study follows 500 mother-child pairs where the mother has a severe hemophilia A gene. It aims to understand bleeding risks during pregnancy and childbirth for mothers, and how children develop antibodies to factor VIII treatment. By observing families over time, researchers …

This study reports on a single adult with severe hemophilia A who is receiving long-term anticoagulation (blood thinners). The goal is to see if newer factor VIII therapies can maintain safe clotting levels while preventing dangerous clots. Only one participant is enrolled, so th…