Blood platelet disease

This early-phase trial tested a combination of two drugs—ruxolitinib and CPX-351—in 12 people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The goal was to find the best dose and see if the combo could shrink cancer. However, the study was stop…

This study tested whether giving the drug caplacizumab based on regular blood tests (ADAMTS13 activity) could improve outcomes for adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare condition causing dangerous blood clots. The trial enrolled 58 adults and mon…

This study tested two extra care programs—supportive palliative care and clinical management of other health issues—for older or frail patients who had a donor stem cell transplant. The goal was to see if these programs could improve quality of life and survival compared to stand…

This study tested a single injection of fibrinogen concentrate followed by a platelet transfusion in blood cancer patients with severe bleeding who did not respond to standard platelet transfusions. The goal was to see if it improved blood clotting. However, the trial was termina…

This study tested a drug called fostamatinib to help people whose blood cell counts drop after a stem cell transplant. The goal was to see if the drug could raise red blood cells or platelets without needing transfusions. The trial was stopped early after enrolling only one perso…

This study tested an experimental drug called VLX-1005 in people with a rare condition where the blood thinner heparin causes a dangerous drop in platelets (cells that help blood clot). Participants received either VLX-1005 or a placebo alongside standard care. The goal was to se…

This study tested a drug called ruxolitinib in 73 people with two types of blood cancer—polycythemia vera or essential thrombocythemia—that were not helped by standard treatment. The goal was to see if the drug could safely reduce high blood cell counts and symptoms like an enlar…

This study tested an experimental drug called iptacopan in people with two rare blood disorders: immune thrombocytopenia (ITP) and cold agglutinin disease (CAD). The goal was to see if the drug could improve platelet counts in ITP or hemoglobin levels in CAD. The trial was termin…

This study tested a drug called RLYB212 in pregnant women at high risk for a condition where the mother's immune system attacks the baby's platelets, causing dangerous bleeding. The goal was to see if the drug was safe and how it moved through the body. Only one woman enrolled be…