Inherited bleeding disorder, platelet-type

This phase IIa trial tests whether FT1, a lab-made version of a natural gut hormone, is safe and helps reduce fluid loss in adults with short bowel syndrome. Participants receive weekly injections of FT1 or a placebo for five weeks. The study measures changes in stool weight and …

This phase 2 trial is testing whether mirabegron, a drug currently used for overactive bladder, can improve glucose tolerance and activate brown fat in people with prediabetes. Researchers will enroll 65 adults with a BMI of 27-45 and prediabetes. Participants will take mirabegro…

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 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 study tests an investigational drug called eptacog beta (SEVENFACT®) in 6 people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. For the first 3 months, participants use their standard treatments for bleeding; then they switch to the study drug to treat se…

This study monitors 40 people in Japan with congenital thrombotic thrombocytopenic purpura (cTTP) who are receiving ADZYNMA (recombinant ADAMTS13) as part of their routine care. Researchers will track side effects and check if the drug improves platelet counts and ADAMTS13 activi…

This study looks at whether giving iron through a vein (IV) can reduce bleeding symptoms in people with inherited bleeding disorders who also have low iron. About 40 participants will receive standard IV iron and be followed for 3 months to see if their bleeding, fatigue, and qua…

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 looks at the real-world safety of Adzynma, a lab-made protein, in people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder. Researchers will review medical records of about 50 children and adults to see how many develop allergi…

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 pilot study from Colorado State University will test 20 healthy adults (ages 18-40) to see if cold temperatures change how the body burns fat during exercise, and whether that change differs between females and males. Participants will exercise in both a normal room and a ve…

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 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 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 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 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…