Hereditary von Willebrand disease

This study tests a new medicine, HMB-002, in about 108 adults with von Willebrand disease (types 1 and 2). The goal is to check safety, how the drug moves in the body, and whether it helps control bleeding. It is the first time this drug is being tested in humans, starting with s…

This study tests a medicine called emicizumab to prevent bleeding episodes in people with Type 3 von Willebrand disease, a severe inherited bleeding disorder. About 75 participants, from infants to adults, will receive either emicizumab or continue their current treatment. The go…

This study looks at whether adding a drug called Wilate (a von Willebrand factor concentrate) to standard platelet transfusions can reduce bleeding and the need for more blood products in intensive care patients. About 120 adults who need platelets during or after surgery will be…

This study tests a drug called vonicog alfa to prevent bleeding in 24 children with severe von Willebrand disease, a condition that causes easy bruising and heavy bleeding. Participants receive the drug regularly for 12 months to see if it reduces bleeding episodes compared to th…

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 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 study looks at whether giving a medicine called Wilate to pregnant women with von Willebrand disease can prevent severe bleeding during and after childbirth. About 110 women will receive Wilate to keep their clotting factor levels high enough during delivery. The goal is to …

This study follows 300 teens and young adults (ages 10-24) with inherited bleeding disorders who choose either a levonorgestrel IUD or norethindrone acetate pills for heavy menstrual bleeding. Researchers will track bleeding patterns, satisfaction, and quality of life over six mo…

This early-phase study tests a new drug called BT200 in 4-6 adults with type 2B von Willebrand disease who have low platelets and a history of bleeding. The drug is designed to raise von Willebrand factor and platelet levels, which may lower the risk of bleeding. Participants rec…

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 program provides continued compassionate access to recombinant von Willebrand factor (rVWF) for children and adults with severe von Willebrand disease who completed a prior study and are benefiting from treatment. Participants must have no comparable alternative therapy avai…

This study is testing a new device that mimics a blood vessel to evaluate how well a person's blood clots under realistic flow conditions. Researchers will compare results from healthy donors, patients with von Willebrand disease, and those on antiplatelet drugs. The goal is to s…

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 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 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 follows 163 teens and young adults with heavy menstrual bleeding, some with a bleeding disorder and some without, to see how well hormonal treatments like the pill, patch, or IUD work. Researchers will measure blood loss, quality of life, and iron levels over time. The…

This study is for people aged 16 to 70 with von Willebrand disease (VWD), a bleeding disorder. Researchers will collect information on bleeding events, treatments, and quality of life over time. The goal is to understand the real-world impact of VWD and use this data as a baselin…

This study creates a web-based registry for people with severe von Willebrand disease (sVWD), a rare bleeding disorder. Researchers will collect information from up to 400 participants about their medical history, symptoms, and lab results. The goal is to build a detailed databas…

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 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 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 creates a registry to collect data on dengue infections in people with congenital bleeding disorders like hemophilia. Dengue can cause serious bleeding, and those with bleeding disorders may face higher risks. The goal is to understand bleeding complications, treatment…

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 looks at 50 adults with unexplained bleeding to see if low serotonin levels in their platelets are linked to their symptoms. Researchers will use a standard bleeding questionnaire to measure bleeding severity. The goal is to better understand how serotonin deficiencies…

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 looks at how blood cells interact and what goes wrong in inherited bleeding disorders like Glanzmann thrombasthenia. Researchers will analyze blood samples from up to 60 healthy volunteers and patients to find genetic defects. The goal is to better understand these con…

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 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…