HEMOPHILIA A

This study tracks adults with hemophilia A who choose to take Jivi, a longer-lasting clotting factor that requires fewer injections. Researchers will use ultrasound to see if joint health stays the same or improves after 24 months of treatment. The goal is to learn how well Jivi …

This study tests a one-time gene therapy called BAY2599023 for adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working copy of the factor VIII gene, so the body can produce its own clotting factor. The trial involves 11 participants and aims to fin…

This phase 3 trial tests a lab-made clotting factor VIII in 60 people with severe hemophilia A. The treatment is given on-demand for new bleeding episodes or before surgery. The goal is to see if it safely and effectively stops bleeding and supports surgical recovery.

This study tests a one-time gene therapy (PF-07055480) in 77 adult men with moderately severe to severe hemophilia A. The goal is to see if the treatment can reduce or prevent bleeding episodes compared to their usual factor VIII infusions. Participants receive a single IV dose a…

This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to help their bodies produce their own clotting factor, reducing or stopping the need for regular factor VIII infusions. Participants will be monitored fo…

This 48-month study observes 427 people with hemophilia A who are already using Elocta to prevent bleeding. Researchers want to see how well the drug protects joints over time, including how many joint bleeds occur and whether target joints improve or worsen. The goal is to under…

This study checks the long-term safety and effectiveness of a one-time gene therapy (BMN 270) for people with severe hemophilia A. Participants from earlier BioMarin trials will be followed for many years to see if the treatment continues to work and remains safe. The goal is to …

This early-stage trial tests a gene therapy for adults with severe hemophilia A who have or had Factor VIII 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 a…

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

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study follows 62 people with hemophilia A who are already using Jivi (a long-acting clotting factor) to prevent bleeding. Researchers will track safety events like side effects and lab changes over at least 4 years during routine doctor visits. The goal is to better understa…

This study looks at how the drug Jivi (damoctocog alfa pegol) works for people with hemophilia A in real-world settings, not just in controlled trials. Researchers are tracking bleeding episodes, side effects, and how satisfied patients and doctors are with the treatment. About 3…

This study looks at whether measuring the amount of emicizumab in the blood can help predict bleeding risk 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. The go…

This study aimed to compare two standard treatments for severe hemophilia A in children: extended half-life factor VIII and non-factor VIII therapies. Researchers planned to measure how well each treatment prevented bleeding and protected joint health using ultrasound. However, t…

This study follows 207 people with hemophilia A who are already taking ADYNOVI/ADYNOVATE to prevent bleeding. Researchers want to see if any long-term side effects appear during normal use. The goal is to gather safety information, not to test if the drug works.