Hemophilia

This early-stage study tests a new drug called SR604 in healthy people and those with hemophilia A, B, or factor VII deficiency. The main goal is to check safety and how the drug moves through the body. About 31 participants will receive single or multiple doses to find the right…

This study looks at whether Emicizumab, a newer drug, prevents bleeding during sports better than standard Factor VIII in children with moderate to severe hemophilia A. About 72 kids aged 6 to 19 who already play sports and take one of these drugs will be followed. The goal is to…

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 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 follows 250 people with hemophilia A who are taking efanesoctocog alfa to prevent bleeding. Researchers want to see how well it protects joints from bleeds over time in everyday life. Participants will be observed for several years to track bleeding rates and treatment…

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 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 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 early-stage trial tests a gene therapy for people with hemophilia A, a bleeding disorder. The treatment uses a modified virus to deliver a working gene into the patient's own stem cells, which are then infused back. The goal is to help the body produce clotting factor VIII, …

This study tests a new gene therapy called SPK-8011QQ in 5 adult men with severe or moderately severe hemophilia A. The goal is to see if it is safe and tolerable. Participants receive a one-time intravenous dose, and researchers monitor for side effects and lab changes.

This trial compares two blood products—prothrombin complex concentrate (PCC) and fresh frozen plasma (FFP)—for controlling bleeding in people undergoing heart valve surgery. Doctors use a special test to guide which product to give and measure how quickly bleeding stops. The goal…

This study tests a single-dose gene therapy called CSL222 (Etranacogene dezaparvovec) in 35 adults with severe or moderately severe hemophilia B who have detectable AAV5 antibodies—a group often excluded from gene therapy. The goal is to see if it reduces bleeding episodes and is…

This program provides concizumab, a daily injection under the skin, for free to people with congenital hemophilia who need better treatment. Participants will be monitored for safety and how well the medicine works over several years. The program is for those with severe hemophil…

This study tests an experimental injection called SR604 in 76 adults with hemophilia A, B, or congenital factor VII deficiency. The goal is to see if it is safe and can reduce bleeding episodes. Participants will receive either a single dose or multiple doses, and researchers wil…

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 study compares a new drug, NXT007, to an existing one (emicizumab) for preventing bleeding episodes in people aged 12 and older with hemophilia A. Participants may have severe, moderate, or mild disease, with or without factor VIII inhibitors. The goal is to see if NXT007 is…

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement th…

This study tests a new drug, NXT007, in people with severe or moderate hemophilia A. The drug is given as a shot under the skin, first every two weeks, then every four weeks. The goal is to see if it is safe and helps control bleeding. About 60 adults, teens, and children will ta…

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 tracks 25 people in Japan with acquired hemophilia A who receive the drug OBIZER to control bleeding. Researchers will watch for side effects and check if the drug helps stop bleeding episodes. The study does not change how doctors treat patients—it simply collects rea…

This study tests an experimental gene insertion therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR/Cas9 to add a working copy of the clotting factor IX gene into the body's DNA, aiming to enable long-term production of…

This study is checking how safe OBIZUR is for adults with acquired hemophilia A, a rare condition that causes sudden and severe bleeding. Researchers will look at medical records and track side effects in about 9 people. The goal is to see how well the drug controls bleeding in e…

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 Nuwiq, a lab-made clotting factor, to prevent excessive bleeding during major surgery in women and girls with hemophilia A. About 28 participants will receive Nuwiq around the time of their surgery. Researchers will check if the drug successful…

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 phase 3 study is testing whether a low, personalized dose of FEIBA (aPCC) can safely control bleeding in children and adults with hemophilia A who have inhibitors and are already taking emicizumab. The study will measure how well the blood clots after FEIBA is given and trac…

This registry follows 100 adults and teens with hemophilia A or B who are prescribed marstacimab, a once-weekly shot under the skin. The main goals are to see if patients prefer this shot over IV treatments and to measure treatment burden after 6 months. Researchers will also tra…

This Phase 1 trial tests a gene therapy that uses a lentivirus to deliver a working clotting factor IX gene into patients' own stem cells. Ten males with severe hemophilia B will receive the modified cells. The goal is to see if it's safe and can raise clotting factor levels, pot…

This study tests an experimental oral medicine called Inno8 in 30 adults with hemophilia A. The goal is to see if it is safe and how the body processes it. Participants will take multiple doses over about 11 weeks. If successful, Inno8 could offer a more convenient way to control…

This study tests whether testicular tissue frozen before puberty can be transplanted back into adult men to restore sperm production. Five men who had fertility-threatening treatments as children will receive their own preserved tissue. The goal is to see if sperm can be found in…

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 study is testing a drug called KN057, given as a weekly injection under the skin, to prevent bleeding in people with hemophilia A or B (with or without inhibitors). The trial will enroll 70 males aged 12 to 65 and will monitor safety and how well the drug works over …

This study looks at whether a treatment called immune tolerance induction (ITI) can help people with haemophilia A who have developed inhibitors—antibodies that block standard clotting factor therapy. Researchers will give high doses of individualized factor VIII concentrates to …

This study is checking whether unlicensed cord blood units are safe to use for stem cell transplants in children and adults with blood disorders. Researchers will closely watch for any infusion-related problems. The goal is to see if these units can be a safe option for patients …

This study tests whether emicizumab (Hemlibra), a medicine given as a shot under the skin, can safely prevent bleeding in people with severe von Willebrand disease (VWD) or combined VWD/hemophilia A. About 40 participants of any age (including infants) will receive the drug and b…

This study tests whether the drug lusutrombopag can help restore platelet levels faster in patients who have had a stem cell transplant for blood disorders. About 45 adults will receive the drug after transplant, and doctors will check platelet counts at 21 days. The goal is to i…

This trial tests a one-time gene therapy called BBM-H803 for adults with severe hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor VIII. The study involves 55 participants and will check safety…

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 follows 500 people with hemophilia B who received HEMGENIX gene therapy, plus a comparison group on standard factor IX treatment. Researchers will track bleeding rates and safety over several years to see if the gene therapy provides lasting control. The goal is to und…

This study tests a medicine called prothrombin complex concentrate (PCC) to stop bleeding episodes in people with hemophilia A who have developed inhibitors (antibodies that block standard treatment). Thirty participants aged 12 to 65 will receive PCC on-demand during bleeding, w…

This study is testing the safety of a drug called SEVENFACT, a clotting factor made from the milk of genetically modified rabbits, in people aged 12 and older with hemophilia A or B who have inhibitors. The drug is already approved for treating bleeding episodes, and this trial w…

This study tests a single-dose gene therapy called GS1191-0445 in 50 Chinese men with hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, helping the body produce its own clotting factor. The goal is to reduce bleeding episodes and the need f…

This study is for adults with blood disorders who are getting a stem cell transplant from a matched donor. Researchers want to see if giving a special potato starch along with a drug that lowers iron (deferasirox) can help prevent serious complications like graft-versus-host dise…

This phase 3 trial is testing a single dose of ANB-002, a gene therapy that delivers a working copy of the FIX gene, in 24 adults with hemophilia B. The goal is to see if it can reduce bleeding episodes compared to regular preventive factor IX infusions. Participants will be foll…

This study tests whether a once-weekly infusion of efanesoctocog alfa can improve joint swelling (synovial hypertrophy) in people with moderate to severe hemophilia A. Researchers will use ultrasound to measure changes in joint health over 12 months. The trial enrolls 35 particip…

This study in Taiwan will follow about 100 people with moderate to severe hemophilia A who are already taking efanesoctocog alfa (a clotting factor) as part of their routine care. Researchers will track joint health, bleeding rates, and safety over up to 5 years using medical rec…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study tests whether a non-invasive light therapy called photobiomodulation can reduce pain and swelling in the knees of children with hemophilia who have joint bleeding. Twenty-four boys and girls aged 9 to 14 will receive the light treatment alongside standard care. Researc…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

This study collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

This study monitors the safety of Jivi (damoctocog alfa pegol) in 20 Korean patients with hemophilia A who have used other treatments before. Researchers will collect data from routine clinic visits over about 8 months per participant, looking for any medical problems. No extra t…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

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 follows 173 people with hemophilia A or B who previously received a single-dose gene therapy in a Pfizer trial. Researchers will track bleeding episodes, clotting factor levels, and any long-term side effects like blood clots or liver issues. The goal is to understand …

This study uses brain MRI scans to look for tiny old bleeds (microbleeds) in 200 adults with severe hemophilia or von Willebrand disease. Researchers want to see how common these microbleeds are and whether they are linked to memory or thinking difficulties. Participants will als…

This study is testing a new, non-invasive method to predict bleeding risk in people with von Willebrand disease and other inherited bleeding disorders. Researchers will use a special microscope to look at tiny blood vessels under the tongue. The goal is to see if the density of t…

This study looks at whether ultrasound and other imaging can find hidden joint changes in people with hemophilia who have no obvious symptoms. Researchers will follow 46 patients for up to 12 weeks, using scans and physical exams to see if these hidden changes are active or inact…

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

This study looks at 36 adults with severe hemophilia A to understand how joint health, pain, balance, and falls impact their quality of life. Researchers will measure joint damage, balance using a special platform, pain levels, and fall history. The goal is to find which factors …

This study is collecting blood and other samples from 5,000 children and young adults (under 21) who are already taking certain medications as part of their regular care. The goal is to understand how these drugs move through and affect children's bodies, so doctors can prescribe…

This study looks at how well hip, knee, shoulder, and other joint replacement surgeries work in adults with inherited bleeding disorders like hemophilia. Researchers will collect information from medical records and follow patients over time to understand risks, complications, an…

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 looks at how gut bacteria and the immune system interact in children and adults receiving stem cell transplants or CAR-T therapy. Researchers will collect stool and blood samples over time to find patterns linked to complications like infections or graft-versus-host di…

This study looks at why people with hemophilia (a bleeding disorder) are less likely to die from heart disease. Researchers will use a new, detailed CT scanner to compare heart artery health in 80 men aged 45+ with hemophilia to similar men without it. The goal is to understand h…

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 is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study follows 102 women who had fertility preservation before cancer treatment to see what happens long-term. Researchers want to know if they get pregnant naturally, use stored eggs or tissue, or decide not to have children. The goal is to improve support and care for femal…

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study is creating a collection of blood samples from 2,000 people with various blood disorders. The samples will be stored and used for future research approved by an ethics board. No treatments or drugs are being tested, and participants continue their normal care.

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…

This study looks at families who carry a change in the DDX41 gene, which may raise the risk of blood cancers like leukemia. Researchers will collect health questionnaires and saliva samples from up to 910 people to track who develops blood problems and when. The goal is to better…

This study looks at using a needle placed into the bone marrow (intraosseous infusion) to give fluids and medicines quickly to critically ill patients with blood diseases. Researchers will enroll 52 adults aged 18-65 to see how often the first attempt works and how safe it is. Th…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This registry collects health data from 4,000 children with hemophilia A or B across Europe. Researchers track bleeding episodes, joint health, and antibody development to clotting factors. The goal is to understand long-term outcomes and improve treatment for this rare bleeding …

This study looks at whether a quick ultrasound of the thigh muscle can detect muscle loss (sarcopenia) in people who have finished cancer treatment. Researchers will compare the ultrasound results with a standard whole-body scan (DEXA) in 55 adults aged 18-74 with certain cancers…

This study follows 120 men with hemophilia A who have developed inhibitors (antibodies that block standard treatment). Researchers will observe how doctors manage their condition using different therapies, including immune tolerance induction and newer drugs like emicizumab. The …

This study is testing new blood tests to see if they can better measure how well bleeding treatments work in people with haemophilia who have developed inhibitors (antibodies that block standard treatment). The researchers will take blood samples from 20 patients before and after…