Inherited bleeding disorder, platelet-type

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 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 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 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 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 It was planned for about 13 months but was withdrawn before enrolling any participants.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…