Blood platelet disease

This study tests whether aerobic exercise in simulated altitude (normobaric hypoxia) can improve body composition and metabolism in people with MASLD, a type of fatty liver disease. Twenty sedentary adults with MASLD will walk on a treadmill for 8 weeks, either in mild altitude o…

This study tests whether a 5-day course of avatricopal can safely raise platelet counts in people with chronic liver disease who have low platelets and need surgery. The goal is to see if more patients reach a platelet count of at least 50×10⁹/L on the day of their procedure, whi…

This early-phase trial tests a new cell therapy called MegaLT for people with dangerously low platelet counts that don't improve after radiation, chemotherapy, or stem cell transplant. Only 10 participants will receive the injection at different doses to check safety and how the …

This study tests whether a low daily dose of the antibiotic linezolid, added to a personalized combination of other drugs, is safe and tolerable for people with multidrug-resistant tuberculosis (MDR-TB). Participants take either linezolid or a placebo for 16 weeks, alongside thei…

This study looked at whether giving romiplostim, a drug that boosts platelet production, could reduce the need for platelet transfusions in patients with low platelet counts after heart or lung transplantation or use of heart/lung support devices. Researchers compared a group of …

This study tested a drug called TAK-755 in 29 adults with a rare blood clotting disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP). The goal was to see if TAK-755, along with standard immune-suppressing drugs, could control the disease with little or no pl…

This study tested a new drug called pelabresib in people with certain blood cancers, including myelofibrosis and leukemia. In the first part, the drug was given alone to find the safest dose. In the second part, it was given with or without another drug (ruxolitinib) to see if it…

This early-phase study tested a new drug called TQ05105 in 79 adults with certain blood cancers (myeloproliferative neoplasms). The main goals were to find the safest dose and understand side effects. The drug works by blocking a protein (JAK2) that can cause abnormal blood cell …

This study tested a medication called avatrombopag in 75 children aged 1 to 17 with immune thrombocytopenia (ITP) lasting at least 6 months. ITP is a condition where the immune system destroys platelets, raising the risk of bruising and bleeding. The goal was to see if the drug c…

This study tested a drug called avatrombopag in 19 Japanese adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The goal was to see if the drug could safely increase platelet counts over 26 weeks. Parti…

This lab study tested whether concizumab, a drug that blocks a natural clotting inhibitor, can improve blood clotting in people with Glanzmann thrombasthenia (GT), a rare genetic bleeding disorder. Blood samples from 20 adults with GT and healthy donors were mixed with concizumab…

This study looked at whether the drug avatrombopag can safely and effectively raise platelet counts in people who develop dangerously low platelets after a liver transplant. Researchers compared 67 patients who received the drug with 67 similar patients who got standard care. The…

This study tested an experimental pill called Bomedemstat in 73 people with essential thrombocythemia, a rare blood condition causing too many platelets. Participants had already tried at least one standard treatment without success. The main goals were to check safety and see if…

This Phase 3 trial tested an oral drug called HMPL-523 (sovleplenib) in 272 adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. Participants who had not responded well to prior treatments received eith…

This study tested a new medicine called HBM9161, given as a weekly shot under the skin, for adults with primary immune thrombocytopenia (ITP) — a condition where the immune system destroys platelets, raising the risk of bleeding. The trial included 36 people who had not responded…

This study tested the drug eltrombopag in 34 people with moderate aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. Eltrombopag is a pill that mimics a natural protein to stimulate blood cell production. Researchers measured whether it safely imp…

This study tested a two-step stem cell transplant for people with blood cancers like leukemia and lymphoma. First, patients received low-dose chemotherapy and radiation to prepare their bodies. Then, they got donor stem cells followed by drugs to prevent the donor cells from atta…

This phase 2 study tested whether the drug romiplostim can prevent severe low platelet counts caused by chemotherapy in people with lymphoma. Eleven adult patients received weekly injections of romiplostim during their 21-day chemo cycles. The goal was to see if it could reduce t…

This phase 3 trial tested the long-term safety and effectiveness of efgartigimod, an IV drug, in 101 adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. Participants received efgartigimod infusions over 52 w…

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

This early-stage trial tested a new drug called PN20 in 24 adults with lymphoma or solid tumors who were getting chemotherapy. The goal was to see if a single injection before chemo could safely prevent a drop in platelets, which can cause bleeding. Researchers checked safety and…

This early-phase study tested a new cell therapy called YTS109 in 7 people with a severe type of autoimmune anemia that did not get better after at least 3 other treatments. The therapy uses specially engineered immune cells to target and destroy the faulty cells causing the dise…

This study tested an experimental oral drug called rilzabrutinib in 86 adults with relapsed immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The drug aims to block a key immune signal to help raise platelet counts. The …

This study tested the drug eltrombopag in 48 Chinese adults with chronic hepatitis B and a bleeding disorder called immune thrombocytopenia (low platelets). The goal was to see if taking eltrombopag for 6 to 22 weeks could safely raise platelet counts. The trial is complete, but …

This study tested two low-dose rituximab schedules in 100 adults with immune thrombocytopenia (ITP), a condition causing low platelet counts and bleeding. The goal was to see which regimen better raises platelets and controls bleeding. Participants had failed or relapsed on stand…

This study tested a medicine called BAX 930 in 52 people born with severe TTP, a rare condition that causes dangerous blood clots. The medicine replaces a missing enzyme to prevent or treat sudden flare-ups. Participants received either BAX 930 or standard treatment for 6 months,…

This phase 2 trial tested the drug sirolimus (rapamycin) in 61 children and young adults with complicated vascular anomalies—abnormal blood vessel growths that can cause pain, bleeding, or organ problems. The goal was to see if sirolimus could safely shrink these anomalies and im…

This study tested whether a drug called caplacizumab, combined with immune-suppressing medicines, could treat a rare blood clotting disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP) without the usual plasma exchange procedure. Fifty-one adults aged 18 to …

This study tested a drug called lusutrombopag in 17 Chinese adults with persistent or chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. Participants had failed standard treatments and took the drug for up to 12 w…

This study tested a drug called eltrombopag in 118 people with bone marrow failure who had a stem cell transplant. The goal was to see if the drug could help their platelet levels recover faster after the transplant. Platelets are blood cells that help stop bleeding, and slow rec…

This study looked at whether taking a protein supplement on fasting days during a 4-week alternate-day fasting program could help young men with overweight lose fat while keeping muscle. Twenty-six men aged 21 to 35 either ate a small meal (400-600 kcal) or the same meal plus a w…

This study tested a doctor-designed dose adjustment algorithm to prevent unplanned delays in FOLFOX chemotherapy for people with cancers of the colon, stomach, esophagus, and other digestive organs. 52 adults participated, and the goal was to see if the algorithm could keep treat…

This early-phase trial tested a new drug called flonoltinib maleate in 31 people with myeloproliferative neoplasms (MPNs), a group of bone marrow cancers. The main goals were to check safety, find the right dose, and see how the body processes the drug. Researchers hope this drug…

This study tested a drug called caplacizumab in 21 Japanese adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare condition causing dangerous blood clots. Participants received caplacizumab alongside standard treatments like plasma exchange and steroids. The goa…

This study looks at the long-term safety of bomedemstat, an oral drug, in 81 people with myeloproliferative neoplasms (MPNs) like essential thrombocythemia and primary myelofibrosis. Participants had already taken bomedemstat in a prior study. Researchers tracked side effects, sp…

This phase 2a trial tested the drug BIVV020 in 12 adults with persistent or chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The main goal was to see if BIVV020 could help maintain a safe platelet count over tim…

This completed phase 3 trial tested whether adding all-trans retinoic acid (ATRA) to eltrombopag works better than eltrombopag alone for adults with immune thrombocytopenia (ITP) that did not respond to steroids. The study enrolled 96 participants and measured sustained platelet …

This phase 2 study tested the drug itacitinib in 4 people with myelofibrosis (a bone marrow disorder) who had already tried other treatments like ruxolitinib or fedratinib. The main goals were to check safety and find the right dose, and to see if it could shrink an enlarged sple…

This study looked at whether treating Helicobacter pylori (a common stomach infection) with antibiotics can raise platelet counts in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding and bruising. Researchers enrol…

This study tested a new diagnostic tool that uses imaging flow cytometry to examine platelets in blood samples. Researchers compared it to standard lab tests in 31 patients suspected of having platelet disorders or heparin-induced thrombocytopenia. The goal was to see if the new …

This study tested a new lab method to diagnose platelet function disorders in 322 people with bleeding symptoms. The method uses flow cytometry to detect signaling defects in platelets, which are often missed by standard tests. The goal was to standardize the technique across mul…

This study tests whether a single injection of nanofat—made from a person's own fat—can improve the appearance of atrophic scars caused by burns or trauma. Each participant has two similar scars: one gets the nanofat injection plus silicone gel, and the other gets only silicone g…

This study tested two oral medications, aprepitant and hydroxyzine, to see which better reduces severe itching triggered by water contact in people with myeloproliferative neoplasms (a type of blood cancer). 63 participants who had persistent itching rated 5 or higher on a 10-poi…

This study is building an international registry of people with GNE myopathy, an ultra-rare muscle disease that causes progressive weakness and often leads to wheelchair use. Participants will complete online questionnaires about their symptoms, medical history, quality of life, …

This study looked at 68 people with low platelet counts who had become resistant to platelet transfusions due to HLA antibodies. Researchers tested a new lab method (C1q binding) on stored blood samples to see if it could better predict whether a transfusion would work compared t…

This study aims to understand how ultra-processed foods affect energy metabolism compared to unprocessed foods. Healthy adults aged 18-60 will stay at a research center for four weeks, eating different diets each week. Researchers will measure how much they eat, their metabolic r…

This study looks back at medical records of people who had an allogeneic stem cell transplant to see whether romiplostim helped their platelets recover faster. Platelets are blood cells that help clotting, and low counts are common after transplant. The researchers will compare p…

This trial investigates whether genetic variations in the CES1 gene influence how the body responds to the blood thinners clopidogrel and aspirin. Healthy Amish adults will take clopidogrel for a week, then a single dose of aspirin, with blood tests measuring platelet function be…

This study followed 78 people with GNE myopathy, a rare genetic disease that causes progressive muscle weakness starting in young adulthood. Researchers collected medical history, blood samples, and muscle function tests over up to two years to understand how the disease progress…

This trial investigates whether a common genetic variation in the CES1 gene affects how the body processes two antiplatelet drugs, clopidogrel and ticagrelor. Healthy volunteers of Amish descent will take each drug for eight days in a crossover design, and researchers will measur…

This study investigates the genetic causes of pediatric Evans syndrome, a rare autoimmune condition where the immune system attacks blood cells. Researchers analyze blood samples from affected children and their families to find known or new mutations. The goal is to better under…

This study investigates inherited thrombocytopenias, rare conditions where low platelet counts cause bleeding problems. About half of patients have an unknown genetic cause. Researchers aim to identify new disease genes and build a lab-grown bone marrow model to test how well dru…

This study follows children in France diagnosed with severe autoimmune cytopenia—conditions where the immune system attacks blood cells. Researchers will track their health over time to see who achieves remission and what factors influence their recovery. The goal is to better un…

This completed study looked at blood samples from 70 children and adults newly diagnosed with immune thrombocytopenic purpura (ITP), a condition that causes easy bruising and bleeding. Researchers wanted to see if certain immune and blood markers could predict whether the disease…

This study looked at 28 people with Glanzmann thrombasthenia, a rare bleeding disorder, to understand why some develop harmful antibodies after blood transfusions or pregnancy. Researchers checked for antibodies every six months and after each transfusion over 18 months. The goal…

This study measured phytoestrogens (plant-based compounds) in the blood and urine of 63 premenopausal women with active lupus, other autoimmune diseases, or no disease. Researchers also asked about diet and measured estradiol levels. The goal was to see if these dietary compounds…

This study looked at whether it is safe to lower the number of platelets needed before placing a central line (a special IV) in patients with blood disorders. Normally, patients get a platelet transfusion if their count is below 50, but this study tested a lower trigger of 10. Th…

This study looked at 68 adults having planned heart surgery that required a heart-lung machine. Researchers compared two different oxygenators (artificial lungs) to see if one caused more damage to platelets, the blood cells that help stop bleeding. They used a special test calle…

This completed study observed 72 pregnant women with HELLP syndrome in the ICU. Researchers tracked daily changes in platelets, liver enzymes, and lactate dehydrogenase before and after delivery. The goal was to understand how these biological markers evolve, not to test a treatm…

This study looked at 166 people with unexplained bleeding to find out how many have problems with tiny granules inside their platelets. These granules help blood clot. The researchers measured granule number, content, and release, and checked if these defects were linked to bleed…

This study followed 37 adults over age 60 with unexplained low platelet counts to see if certain tests could predict immune thrombocytopenic purpura (ITP). Participants had blood tests, bone marrow biopsies, and a short course of the steroid prednisone. The goal was to find which…

This study followed over 14,000 pregnant women to see how many are at risk for a condition called FNAIT, where a mother's immune system attacks her baby's platelets, causing dangerous bleeding. Researchers measured how often women develop specific antibodies (HPA-1a) and looked a…

This study looked at how a pill form of THC (dronabinol) affects sleep and thinking in 18 adults. Some participants used cannabis frequently (more than 3 times a week), while others rarely used it (less than 10 times ever). Researchers measured sleep stages, alertness, memory, an…

This study looked at 33 adults who had both immune thrombocytopenia (ITP) — a condition where the immune system destroys platelets — and cancer. Researchers wanted to see if treating the cancer could also improve ITP. They also investigated whether cancer cells might mimic platel…

This study looked at how chronic immune thrombocytopenia (ITP) affects thinking skills and fatigue in adults. Researchers measured brain function and inflammation using blood tests and questionnaires in 97 patients. The goal was to better understand these connections, not to test…

This study tested whether a blood test called thromboelastography (TEG) can better guide blood-thinning treatment in 61 patients on ECMO life support. ECMO patients face serious risks from bleeding or clotting, and current monitoring methods are not very reliable. Researchers com…

This study looked at 50 adults with severe low platelet counts who needed platelet transfusions. Researchers wanted to understand why some patients stop responding to these life-saving transfusions. They focused on antibodies that attack the donor platelets, aiming to find better…

This completed Phase 2 study looked at 244 glioblastoma patients to see if platelet counts during radiation and chemotherapy could predict who would develop dangerously low platelets later. The goal was to create a monitoring plan for high-risk patients. No new drug or treatment …

This study looked at how well a daily low-dose aspirin works in people who have both immune thrombocytopenia (low platelets) and heart disease. Only 4 adults took part. Researchers measured platelet function after aspirin to see if the standard dose is enough to prevent blood clo…

This study checked whether a generic eltrombopag olamine tablet works the same as the brand-name version in 36 healthy Chinese adults after a meal. Participants took a single dose of each formulation in random order, and researchers measured how the drug moved through their bodie…

This completed registry study followed 80 children and young adults with severe immune cytopenias, including immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome. Researchers observed participants over up to 4 years to improve diagnosis and track the disease c…

This completed pilot study looked at whether some healthy adults are resistant to aspirin's blood-thinning effects. 48 participants took two different types of 81 mg aspirin for two weeks each, and their platelet function was measured. The goal was to understand how common aspiri…

This study looked back at 223 people with acquired thrombotic thrombocytopenic purpura (aTTP) who received caplacizumab (Cablivi). The drug quickly normalizes platelet counts, but some patients experience a sharp rise then a dip, which might mislead doctors into giving unnecessar…

This study looked at data from over 1000 preterm babies with low platelet counts to build a model that predicts their risk of major bleeding or death. The goal is to help doctors decide when a platelet transfusion is truly needed, since current guidelines based only on platelet c…

This study collected one blood sample from 40 adults with very low platelets (30,000 or fewer per microliter) due to blood cancers or their treatments. The goal was simply to gather samples for research, not to test any new drug or therapy. Participants had to be 18 or older and …