Coagulation protein disease

This study tests a daily injection called concizumab in children under 12 with hemophilia A or B, with or without inhibitors. The goal is to see if it safely prevents bleeding episodes. Participants receive daily shots under the skin for at least 2 years, and the study tracks how…

This study tests a medicine called concizumab for people with hemophilia A or B who do not have inhibitors. The goal is to see if a daily shot under the skin can prevent bleeding episodes safely. About 156 participants, aged 12 and older, will either start concizumab right away o…

This observational study compares the safety of two drugs, BALFAXAR and KCENTRA, used to reverse the effects of vitamin K antagonist blood thinners (like warfarin) in adults who need urgent surgery or an invasive procedure. The main goal is to see how often blood clots (thromboem…

This study tests Mim8, a drug that helps blood clot by mimicking a missing protein, in people with hemophilia A (with or without inhibitors). Over 450 participants will receive injections for up to 5.5 years to see if it safely prevents bleeding episodes. The goal is to provide a…

This study tests a drug called concizumab, given as a daily shot under the skin, to prevent bleeding in people with hemophilia A or B who have inhibitors (antibodies that block standard treatment). About 134 participants either start concizumab right away or continue their usual …

This phase 3 trial tests a new drug called FRSW107, a lab-made clotting factor VIII fused with a protein that helps it last longer in the body. The study enrolls males aged 12 to 65 with severe hemophilia A who have had at least 150 prior exposure days to factor VIII products. Pa…

This phase 3 trial tests a new drug called FRSW107, a long-lasting version of clotting factor VIII, in people with severe hemophilia A. The study enrolls males aged 12 to 65 who have had at least 150 prior exposure days to factor VIII products. Participants receive regular inject…

This study tracks the safety of Hympavzi (marstacimab) in 50 people with hemophilia A or B who do not have inhibitors. Participants receive weekly injections to help control bleeding. The main goal is to monitor any side effects in real-world use.

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 Phase 3 trial is testing STSP-0601, an injectable drug, to control bleeding episodes in people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The study will enroll 40 participants aged 12 to 70 and measure how well the drug s…

This phase 3 trial is investigating the long-term safety and effectiveness of an experimental clotting factor called BIVV001 in people with severe hemophilia A. The study aims to see if regular infusions of this therapy can prevent bleeding episodes, protect joint health, and imp…

This Phase IV study is testing emicizumab (Hemlibra) in 136 people aged 13 to 70 with moderate or severe hemophilia A who do not have inhibitors. The goal is to see if this drug, given as a shot under the skin, can improve joint health, increase physical activity, and enhance ove…

This trial tests whether applying tranexamic acid (TXA) directly to the face during facelift surgery can reduce bleeding and bruising better than a placebo. TXA is a medicine that helps blood clots stay intact, and it is already used intravenously for heavy bleeding. Here, it is …

This study looks at how well Eqwilate, a concentrated mix of two clotting proteins (VWF and FVIII), controls bleeding in people with von Willebrand disease. It includes patients aged 6 and older who use Eqwilate on demand, before surgery, or as regular prevention. Researchers tra…

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 is testing a medicine called emicizumab (Hemlibra) in babies from birth to 12 months old with severe hemophilia A. The goal is to see if a weekly or biweekly shot under the skin can prevent bleeding episodes and protect joints. About 55 infants will receive the drug fo…

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 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 phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…

This study follows 30 people with hemophilia A or B (without inhibitors) in Japan who are already using Alhemo (concizumab) as part of their routine care. Researchers will monitor side effects and how well the drug works over about 2 years per person. The goal is to see if Alhemo…

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 for adult men with moderate to severe hemophilia B, a bleeding disorder. The therapy delivers a working gene to help the body produce a clotting protein called Factor IX. Researchers will track how many bleeding episodes participants have …

This study was designed to test how FANHDI, a clotting factor concentrate, works in children under 6 with severe von Willebrand disease (types 2 or 3). The goal was to measure how the drug moves through the body and how well it controls bleeding. However, the trial was withdrawn …

This early-stage trial is testing a single-dose gene therapy called GS1191-0445 in 7 Chinese adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor. The main goal is to check safet…

This study tests whether a once-weekly medication (efanesoctocog alfa) can improve or prevent joint damage in 37 people with moderate or severe hemophilia A. Participants receive the drug for 12 months, and doctors use ultrasound and MRI to check joint health. The goal is to see …

This phase 3 trial is testing a new form of factor VIII (rVIII-SingleChain) in 60 Chinese men with severe hemophilia A. The goal is to see how well the drug works to prevent and treat bleeding episodes when given 2-3 times per week. Researchers will also check how the body proces…

This clinical trial is testing a one-time gene therapy called BBM-H901 for people with hemophilia B, a bleeding disorder caused by low levels of clotting factor IX. The therapy uses a harmless virus to deliver a working copy of the factor IX gene to the liver, so the body can pro…

This study tests a single intravenous dose of a gene therapy called AskBio009 in up to 30 adults with severe hemophilia B. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The main goal is to check safety, but researchers…

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 looks at whether switching to low-dose emicizumab can improve joint health and reduce bleeding in people with hemophilia A who still have joint problems despite standard factor VIII therapy. Eighteen patients aged 8-28 from Thailand were enrolled. Those with worse join…

This phase 3 trial tests fitusiran, an injectable drug, in 91 males aged 12 and older with severe hemophilia A or B, with or without inhibitors. Participants first receive standard care for 6 months, then switch to fitusiran for about 3 years. The goal is to see if fitusiran redu…

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 study follows about 60 males with hemophilia A who are already using Esperoct to prevent bleeds. Researchers will track side effects and how well the medicine controls bleeding over 5-7 years. Participants continue their normal treatment and clinic visits, with no extra proc…

This study looks at the long-term safety and effectiveness of a drug called fitusiran in people with hemophilia A or B, including those with inhibitors that make standard treatments less effective. The main goal is to see if fitusiran is safe over time and if it reduces bleeding …

This phase 2 trial tests the drug emicizumab (Hemlibra) as a regular injection to prevent bleeding in 51 adults with acquired hemophilia A, a rare condition where the immune system attacks clotting factors. Participants receive two loading doses then weekly shots, along with stan…

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 Phase 3 trial tests a new drug called KN057, given as a weekly injection, to prevent bleeding in people with hemophilia A or B who do not have inhibitors. The study includes 125 males aged 12 to 70 with severe or moderately severe hemophilia. Participants are split into grou…

This study tests a weekly injection called KN057 to see if it can prevent bleeding episodes in people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). About 53 male participants aged 12 to 70 will be randomly assigned to receive KN0…

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 tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study tests whether a training method that briefly restricts blood flow to the leg muscles can help reduce knee pain in people with hemophilia. Twelve participants will try both the real blood flow restriction and a sham version to compare effects. The goal is to see if this…

This study observes 44 people with type 3 von Willebrand disease, a rare bleeding disorder, who are already on standard preventive treatment. Researchers will track how often bleeding episodes occur and how the condition affects quality of life over at least 24 weeks. No new drug…

This study follows 11 males with Hemophilia B who already receive regular factor IX therapy to prevent bleeding. Researchers will collect information on bleeding events, treatment use, and quality of life over at least 6 months. No experimental treatment is given; the goal is to …

This study evaluates whether artificial intelligence models can accurately interpret ROTEM blood clotting tests and recommend treatments for coagulopathy. Researchers will compare AI decisions to those of a panel of clinical experts using data from adults undergoing elective card…

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 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 tracks the safety and effectiveness of the drug Alhemo (concizumab) in 23 people with hemophilia A or B in Japan. Participants receive the drug as part of their normal care, and researchers will monitor side effects for about 2 years. The goal is to see how well Alhemo…

This study follows 60 males with haemophilia B who are already using the drug Refixia (nonacog beta pegol) to prevent bleeding. Researchers will collect information on side effects, bleeding episodes, and quality of life over 4 to 9 years. No new treatment is given—participants c…

This study surveys 350 adults with hemophilia to better understand chronic pain—its type, location, and impact on daily life. Participants fill out questionnaires about their pain history, medication use, and emotional well-being. The goal is to help doctors recognize and treat p…

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 looks at whether adding simple family history questions to a standard bleeding questionnaire can better identify children who truly have mild bleeding disorders. Researchers will review data from 1,000 children seen at a clinic since 2019. The goal is to see if this co…

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 is observing 500 male patients aged 12 and older with severe hemophilia A or B across China. Researchers are collecting data on treatment patterns, costs, and quality of life to understand the true burden of the disease. The goal is to identify gaps in care and help im…

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.

This study collects information from doctors and patient groups across Asia and the Pacific to better understand how haemophilia affects people in the region. No treatments or medications are given; instead, researchers will analyze data on how many people have the condition, how…

This study gathers safety information from a European registry for people with haemophilia A who are treated with turoctocog alfa pegol. Researchers will count any side effects reported to the registry that might be linked to the drug. About 50 participants are involved, and ther…