Inherited blood coagulation disorder

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 fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps preven…

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 donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

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 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 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 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 whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants receive the drug and are monitored for side effects and changes in blo…

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 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 phase 2 study tests two drugs, bimagrumab and tirzepatide, alone or together, to see if they help adults with obesity or overweight lose weight safely. About 252 participants without type 2 diabetes will take the drugs or a placebo for up to 70 weeks. The main goal is to mea…

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 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 gene therapy (PF-06838435) for people with severe hemophilia B, a bleeding disorder. The therapy uses a harmless virus to deliver a working gene for clotting factor IX. Researchers are following 21 participants long-term to see if it is safe and reduces bleedin…

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 a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

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 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 study tests a new medicine called crovalimab for people with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots and kidney damage. The trial includes 83 adults and teens who receive the drug to see if it can control the disease and impr…

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 phase 2 trial tests whether the drug itacitinib, given with standard care, can prevent graft-versus-host disease (GVHD) in people who receive a donor stem cell transplant for blood cancers. GVHD happens when the donor cells attack the patient's body. The study includes 31 ad…

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 medicine called TAK-755 in people with a rare inherited blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP). The condition causes dangerous blood clots and bleeding. TAK-755 replaces a missing enzyme to help prevent or treat these episod…

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 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 phase 3 trial tests crovalimab in 41 children with atypical hemolytic uremic syndrome (aHUS), a rare disease that causes blood clots and kidney damage. The drug is given first through a vein, then as a shot under the skin every four weeks. The goal is to see if it can stop d…

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 study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…

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 phase 3 trial is testing whether liver surgery followed by two drugs (atezolizumab and bevacizumab) can help people with liver cancer that has spread into veins. The study includes 501 participants who have not had prior treatment. The goal is to see if this approach delays …

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 phase 3 trial tests a long-acting clotting factor (rIX-FP) in 23 Chinese men with severe hemophilia B. The drug is given regularly to prevent bleeding episodes. Researchers will measure how well it works, how long it lasts in the body, and check for side effects like allergi…

This early-phase trial tests an experimental antibody called HMB-001 in 57 adults with Glanzmann thrombasthenia, a rare bleeding disorder. The study aims to see if the drug is safe and can reduce the number and severity of bleeds when given regularly. Participants receive single …

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 tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…

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 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 Phase II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to …

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 trial tests a gene therapy called OTL-103 for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The therapy uses the patient's own blood stem cells, modified in a lab to carry a working copy of the faulty gene, …

This study aimed to test two drugs, bimagrumab and tirzepatide, alone or together, for helping adults with obesity or overweight and type 2 diabetes lose weight. It was planned for about 13 months but was withdrawn before enrolling any participants. The main goal was to see how m…

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 pilot study tests whether a daily mindful music listening program is practical and acceptable for patients hospitalized after an allogeneic stem cell transplant. About 35 adults will listen to music for one hour each day using a web-based platform. The study measures how fea…

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 looks at whether adding art therapy to physical therapy can help children aged 5 to 18 recover emotionally and physically after a stem cell transplant. For two weeks, some children receive both art and physical therapy daily, while others get only physical therapy. Res…

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 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 collects and analyzes blood, DNA, and tissue samples from 315 adults with pulmonary fibrosis, their relatives, and healthy volunteers. The goal is to learn more about the disease's causes, not to test a new treatment. Researchers hope the findings will guide future the…

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 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 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 aimed to collect medical information from people who developed unusual blood clots within four weeks of receiving a COVID-19 vaccine. The goal was to better understand any possible link between the vaccine and these rare events. However, the study was withdrawn before …

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 follows 50 young men who had cancer or blood disorders as children and received treatments that can harm fertility. Some stored testicular tissue before treatment; others did not. Researchers will check their fertility through exams, ultrasounds, blood tests, and semen…

This study observes 367 Chinese patients with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. Researchers will track how patients are treated in routine care and monitor their health for up to 12-24 months. The goal is to bet…

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 observational study will follow 384 patients in Brazil who are using cannabis-based products (like CBD or THC oils) prescribed by their own doctors for chronic pain, anxiety, or depression. Researchers will not provide the medication but will track patients' quality of life,…

This study aims to develop better lab tests to diagnose and monitor two rare kidney diseases: atypical hemolytic uremic syndrome (aHUS) and C3 glomerulopathy (C3G). Researchers will analyze blood, cells, and kidney tissue from 180 patients and healthy volunteers to find markers t…

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 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 looks at what causes dangerous blood clots in the legs (venous thrombosis) and lungs (pulmonary embolism) in over 21,000 African American and white adults. Researchers are using data from two long-running heart health studies to find patterns and risk factors. The goal…

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…