Blood platelet disease

This study tests a new biologic drug called ianalumab combined with a standard platelet-boosting drug (TPO-RA) in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial includes 164 adults who have tried at l…

This trial tests whether sirolimus (rapamycin), an immune-suppressing drug, can safely raise platelet counts in people with antiphospholipid antibodies and persistent low platelets. Participants are randomly assigned to receive sirolimus or a placebo for six months, and those who…

This phase 3 trial compares bomedemstat, an experimental oral drug, to hydroxyurea (standard therapy) in 300 people with essential thrombocythemia who have not yet received cytoreductive treatment. The goal is to see if bomedemstat better controls platelet counts and prevents com…

This study tests a drug called pirtobrutinib in adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. The trial has two parts: first, finding safe doses, then comparing pirtobrutinib to a placebo to see if it ra…

This study tests an oral drug called rilzabrutinib in adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. Participants must have failed first-line treatment. The goal is to see if the drug can raise platelet c…

This study tests if the drug sirolimus can help adults with lupus who also have very low platelet counts (a condition called immune thrombocytopenia). Participants will receive either sirolimus or a placebo for 24 weeks, then all will receive sirolimus for another 24 weeks. The g…

This study tests a drug called tafasitamab in adults with two rare autoimmune blood disorders: immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA). The goal is to see if it can safely raise blood cell counts and reduce the need for other treatments. About 5…

This study tests a medicine called KIg 10 (intravenous immunoglobulin) in adults with chronic ITP, a condition where the immune system destroys platelets, causing bleeding and bruising. The goal is to see if KIg 10 can safely raise platelet counts and stop bleeding. About 40 peop…

This Phase 2 trial tests STP938, a daily pill, in 50 adults with high-risk essential thrombocythaemia who cannot use standard therapy. The goal is to see if it safely lowers high platelet counts and reduces complications like blood clots. Participants take the drug for up to 12 m…

This early-stage trial tests a one-time treatment called CTX112, made from donor immune cells edited with CRISPR technology. It aims to safely control two autoimmune diseases where the body attacks its own blood cells: immune thrombocytopenia (ITP) and warm autoimmune hemolytic a…

This Phase 3 trial tests whether mezagitamab, given as a shot under the skin, can safely keep platelet counts stable in adults with chronic ITP. ITP is a condition where the immune system destroys platelets, leading to easy bruising and bleeding. The study will compare mezagitama…

This early-stage study tests a new treatment called RN1201 for people with certain blood disorders caused by the immune system attacking the body's own cells. The treatment involves giving patients specially designed immune cells after a short course of chemotherapy to weaken the…

This pilot study tests efgartigimod, a drug that lowers harmful antibodies, in 15 adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare and life-threatening blood disorder. Participants receive weekly infusions for 8 weeks, then are monitored for another …

This study looks at 300 adults with low platelet counts who need a heart stent. It compares two common blood thinner combinations (aspirin plus clopidogrel vs. aspirin plus ticagrelor) to see which causes fewer bleeding problems. The goal is to find the safer option for these pat…

This study tests an IV drug called efgartigimod in teens aged 12 to 18 with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bruising and bleeding. The goal is to find the right dose and see if it safely raises platelet counts…

This study tests whether a second course of ianalumab can help adults with two rare blood disorders—immune thrombocytopenia (ITP) and warm-antibody autoimmune hemolytic anemia (wAIHA)—who previously responded to the drug but then stopped responding. About 60 participants will rec…

This early-stage study tests a new drug called JNJ-88549968, a bispecific antibody designed to direct the body's immune T cells to attack cancer cells with a specific CALR mutation. The trial will enroll about 220 adults with essential thrombocythemia or myelofibrosis who carry t…

This study tests whether a drug called nipocalimab can prevent severe bleeding in unborn babies at risk of FNAIT, a condition where the mother's immune system attacks the baby's platelets. About 50 pregnant women with a history of FNAIT will receive either nipocalimab or standard…

This study tests the long-term safety of a drug called mezagitamab for adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing easy bruising and bleeding. About 150 participants who completed earlier mezagitamab studies w…

This study tests a medicine called nipocalimab to see if it can lower the risk of severe bleeding or death in unborn babies with a rare condition called FNAIT. The study includes 39 pregnant women who have had a previous pregnancy affected by FNAIT. Participants receive either ni…

This study tests a new drug called NVG-2089 in 30 adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. The drug is designed to work like IVIg, calming the immune system. Researchers will check for side effects …

This study tests a new daily pill called JBI-802 in people with certain blood cancers (myeloproliferative neoplasms and MDS/MPN) who have too many platelets. The trial has two phases: first, finding a safe dose in about 30 adults, then checking if it works. Participants take the …

This phase 2 trial tests a bone marrow transplant using lower doses of chemotherapy and radiation to prepare the body, along with a drug called cyclophosphamide after transplant to prevent graft-versus-host disease. It enrolls 60 people with severe aplastic anemia or related bone…

This early-stage study tests an experimental drug, INCA036978, in about 218 adults with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check the drug's safety and find the best dose, either alone or with standard treatment. Participants must have a l…

This early-stage trial is testing a new drug called IASO-782 in 16 adults with autoimmune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA). These conditions cause the immune system to mistakenly attack blood cells. The study's main goal is to check the drug's sa…

This early-stage trial is testing a new drug called SHR-2173 for people with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The study will enroll 10 adults to check the drug's safety and how well it works. Particip…

This study tests a weekly injection called romiplostim in 60 adults with immune thrombocytopenia (ITP) whose platelet counts stayed low despite oral medications. The goal is to see if the shot can safely raise platelet levels and reduce bleeding over 24 weeks. Participants stop t…

This early-stage trial tests an injectable drug called GL-2045 in 42 adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The goal is to see if GL-2045 can safely raise platelet counts like standard IVIg therapy…

This study tests whether adding all-trans retinoic acid to romiplostim N01 can better raise platelet counts in adults who have low platelets after finishing chemotherapy for gastrointestinal cancers. About 220 participants will receive either the combo or romiplostim alone for 12…

This study is for adults with certain cancers (like breast, lung, or ovarian) that are in complete remission but have persistently low platelet counts from past chemotherapy. It tests whether adding a vitamin A derivative (ATRA) to a standard platelet-boosting drug works better t…

This study tests an experimental drug called PF-06835375 in adults with moderate to severe primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The goal is to see if the drug can safely increase platelet counts. Abo…

This study tests whether a single infusion of umbilical cord blood can safely raise platelet levels in cancer patients whose platelets have dropped due to chemotherapy or other cancer treatments. Researchers will enroll 25 people aged 12 to 65 and monitor their blood counts for 2…

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 early-phase study tests a new drug called INCB160058 in adults with myeloproliferative neoplasms, a group of rare blood cancers. The main goal is to check the drug's safety and how the body processes it. About 186 participants will receive the drug to find the right dose and…

This study tests an investigational IV drug called efgartigimod in adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. About 69 participants will receive either the drug or a placebo for up to 24 weeks,…

This study tests an experimental drug, hetrombopag, to see if it can safely raise platelet counts in cancer patients whose platelets drop too low after chemotherapy. About 150 adults with solid tumors (like lung, breast, or colon cancer) will receive either the drug or a placebo …

This study tests whether adding orelabrutinib to high-dose dexamethasone works better than dexamethasone alone for adults newly diagnosed with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets. About 86 participants will receive either …

This study tests whether adding rituximab to eltrombopag works better than eltrombopag alone for adults with immune thrombocytopenia (ITP) whose platelet counts stay low despite prior treatments. About 224 participants will receive either the combo or eltrombopag alone, and resea…

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 early-stage trial tests a new drug called INCA033989, alone or with an existing drug (ruxolitinib), in people with myeloproliferative neoplasms—a group of rare blood cancers that cause the bone marrow to produce too many cells. The study aims to find safe doses and check for…

This study is collecting information from 40 patients with a rare blood disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP) who have been treated with daratumumab. The goal is to see how well daratumumab works and how safe it is for patients whose disease d…

This study tests whether a specially adjusted dose of the blood thinner apixaban can safely treat blood clots in cancer patients who also have low platelet counts. About 30 adults with active cancer and a recent clot will receive the adjusted dose. The goal is to see if this appr…

This study tests a new drug called romiplostim N01, given as a weekly shot, against a standard daily shot (rhIL-11) for cancer patients whose platelet counts drop due to treatment. About 88 adults with various cancers and low platelets will be randomly assigned to one of the two …

This program offers eltrombopag to patients with serious or life-threatening thrombocytopenia (low platelet counts) who have no other treatment options and cannot join a clinical trial. A licensed physician must request the drug, and the potential benefit must outweigh the risks.…

This study tests a stronger, more frequent dosing schedule of the drug rituximab in children and young adults (ages 1 to 21) with newly diagnosed immune thrombocytopenic purpura (ITP) who are at high risk for severe bleeding. The goal is to see if this approach can safely improve…

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 Phase 3 trial tests a human immunoglobulin (IVIG) given intravenously for 5 days to adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risk. The study will enroll 36 participants in Turkey to see if the…

This early-stage trial tests a new drug, INCA035784, in 120 adults with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check safety and find the right dose. Participants must have a specific genetic mutation (CALR) and have tried other treatments bef…

This study tests a drug called romiplostim to see if it can safely increase platelet counts in people with low platelets before they have heart surgery. Low platelets can make surgery risky, and this treatment aims to reduce the need for blood transfusions. About 136 adults sched…

This early-stage trial is testing a drug called RAY121 in 144 people with one of six immune diseases, including antiphospholipid syndrome and bullous pemphigoid. The drug works by blocking a specific part of the immune system (the complement pathway) that can cause tissue damage.…

This study follows about 400 people with certain blood cancers (like essential thrombocythemia and myelofibrosis) who are already taking bomedemstat and benefiting from it. The goal is to collect more information on the drug's long-term safety and how well it controls the disease…

This early-phase trial is testing a new drug called budoprutug (TNT119) in 24 adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. Participants receive two IV doses and are monitored for safety, how the drug mov…

This phase 2 trial is testing a new approach for people with a specific type of rectal cancer that has spread to a few other spots in the body (oligometastases). The treatment combines short-course radiotherapy, chemotherapy (CAPEOX), and an immunotherapy drug called serplulimab …

This study looks at whether giving platelet transfusions only when levels are very low can help extremely premature babies (born at 23-26 weeks) survive without major bleeding. About 2,400 infants will be randomly assigned to a lower or higher transfusion trigger. The goal is to …

This early-stage trial is testing a new drug called INCA033989, alone or with an existing drug ruxolitinib, in 225 people with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check safety and find the best dose. Participants must have a specific genet…

This study tests the drug luspatercept in 50 adults with clonal cytopenias of uncertain significance (CCUS), a condition where low blood cell counts increase the risk of developing blood cancers like leukemia. Participants receive injections every three weeks for up to 48 weeks. …

This phase 2 trial is testing whether the drug hetrombopag can prevent chemotherapy-induced thrombocytopenia (low platelet counts) in people with gastrointestinal tumors. The study will enroll 40 patients who had low platelets in a previous chemo cycle. Participants take hetrombo…

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 program provides early access to the drug ianalumab (VAY736) for patients with serious or life-threatening conditions like immune thrombocytopenia or Sjögren's syndrome who have no other treatment options. It is not a clinical trial but a managed access program where doctors…

This phase 3 trial is testing whether direct oral anticoagulants (DOACs) like apixaban or rivaroxaban are better than low-dose aspirin at preventing blood clots in people with JAK2V617F-positive myeloproliferative neoplasms (blood cancers). The study plans to enroll 1308 high-ris…

This study tests whether giving a chemotherapy drug (decitabine) plus a targeted therapy (ruxolitinib, fedratinib, or pacritinib) before a stem cell transplant helps people with advanced myeloproliferative neoplasms (MPN) get to transplant and have better outcomes. About 25 adult…

This study tests a new treatment using a patient's own immune cells, modified to target and attack faulty immune cells that cause autoimmune diseases. It is for people with severe, hard-to-treat conditions like lupus. The goal is to see if this therapy is safe and can control the…

This study tests whether starting two oral medications (cyclosporine and eltrombopag) earlier than usual is safe and feasible for people with severe aplastic anemia, a rare condition where the immune system attacks bone marrow. Participants aged 3 and older will take these pills …

This early-stage study tests a new cell therapy called YTS109 in 18 adults aged 18-65 with autoimmune diseases like lupus and Sjogren's syndrome that have not responded to standard treatments. Participants receive a single infusion of the cells, and researchers monitor safety and…

This early-stage trial is testing a new drug called PN20 in 12 adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. Participants receive a single injection of PN20, which is designed to stimulate platele…

This early-stage study tests a new treatment called RD06-05, a type of CAR-T cell therapy, in 27 adults with autoimmune blood diseases like immune thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA), or Evans syndrome. The therapy aims to control the disease by targeting a…

This study tests whether etamsylate, a drug that helps blood clot, can prevent serious bleeding in people who have low platelets after a stem cell transplant. About 404 adults will receive either etamsylate or a placebo, and researchers will track who has a major bleed or dies wi…

This early-stage trial is testing a new treatment called YTS109 for people with severe autoimmune diseases like lupus and Sjogren's syndrome that have come back or not responded to standard care. About 18 adults will receive a single infusion of these specially engineered immune …

This early-stage study tests a new cell therapy called YTS109 in 18 adults with severe autoimmune diseases like lupus and Sjogren's syndrome that have not responded to standard treatments. Participants receive a single infusion of the cells, and researchers monitor safety and how…

This early-stage study tests a new cell treatment called YTS109 in 18 adults with hard-to-treat autoimmune diseases like lupus and scleroderma. Participants receive a single infusion of these specially designed cells. The main goal is to check if the treatment is safe, and also t…

This study tests a single injection of PN20, a lab-made protein, to see if it can safely raise platelet counts in adults with chronic liver disease who have low platelets and are scheduled for a minor procedure. About 54 participants will receive either PN20 or a placebo. The mai…

This study tests whether a simpler treatment for immune thrombotic thrombocytopenic purpura (iTTP) works as well as the standard approach. Instead of daily plasma exchange (an invasive procedure), patients receive plasma infusions, immune-suppressing drugs, and caplacizumab. The …

This Phase 2 trial tests the drug selinexor in people with myelofibrosis who have not taken JAK inhibitors and have low to normal platelet counts. The main goal is to see if selinexor can shrink the spleen by at least 35% after 24 weeks. Researchers will also check if it improves…

This study compares two antibody drugs—daratumumab and rituximab—in 160 adults with immune thrombocytopenia (ITP) whose condition did not improve or came back after steroid treatment. The goal is to see which drug works better at raising platelet counts and reducing bleeding risk…

This study tests whether adding an anti-CD38 antibody to the standard drug rituximab works better than rituximab alone for adults with immune thrombocytopenia (ITP) whose platelet counts stayed low or came back after steroid treatment. About 160 participants will receive either t…

This study tests a new drug called elritercept (KER-050) for people with myelofibrosis, a rare bone marrow disorder. The drug is given alone or with the standard treatment ruxolitinib. The main goals are to check safety and see if it helps with anemia and other symptoms. About 13…

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 phase 2 trial tests the drug bortezomib in 29 adults with relapsed or refractory immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. Participants receive bortezomib injections over three weeks for three cycles, then a…

This study tests a new drug, daratumumab, against an older one, rituximab, in children aged 6 to 17 with immune thrombocytopenia (ITP) that didn't improve with steroids. ITP causes low platelets and easy bleeding. The goal is to see which drug works better at raising platelet cou…

This phase 2 trial tests whether combining rituximab with daratumumab can raise platelet counts in adults with immune thrombocytopenia (ITP) who have not responded to steroids. Twenty participants will receive one dose of rituximab and eight doses of daratumumab over eight weeks.…

This early-stage study tests a new treatment called RXIM002 for people with severe autoimmune diseases that haven't responded to standard therapies. The treatment uses a special RNA molecule to create immune cells that target and destroy faulty B cells, which are causing the dise…

This phase 2 trial tests an experimental oral drug called DZD8586 (Birelentinib) in adults with primary immune thrombocytopenia (ITP) whose condition did not improve after at least one standard therapy. About 60 participants will receive one of three dose levels to see if it safe…

This early-stage study is testing a new type of cell therapy (called QT-019C) in 12 adults with severe autoimmune diseases like lupus and scleroderma that have not responded to standard treatments. The therapy uses specially engineered immune cells from a healthy donor to target …

This study tests whether V-IMMUNE, a human immunoglobulin given through a vein, can safely raise platelet counts in people with immune thrombocytopenia (ITP). About 31 adults and children aged 1 year and older with very low platelets will receive two daily infusions. The main goa…

This early-phase trial tests a new drug called STSA-1301, given as an injection under the skin, for people with immune thrombocytopenia (ITP) — a condition where the immune system destroys platelets, raising bleeding risk. The study will first check safety in healthy volunteers, …

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study tests an experimental drug called CM336 in people with relapsed or refractory autoimmune cytopenia, including ITP and autoimmune hemolytic anemia. CM336 is a bispecific antibody designed to target and eliminate certain immune cells. The trial will enroll 158 adults to …

This study tests low-dose methotrexate, a common arthritis drug, in 54 people with three rare blood cancers: polycythemia vera, essential thrombocythemia, and myelofibrosis. Researchers want to see if it can shrink spleens, improve symptoms, and lower blood counts safely. If succ…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

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 phase 2 trial is testing a drug called Hetrombopag to raise platelet counts in 56 breast cancer patients who developed low platelets (thrombocytopenia) after T-DM1 therapy. The goal is to see if the drug can safely keep platelets at a safe level so patients can continue thei…

This study tests a combination of two drugs, romiplostim and rituximab, in about 30 adults with primary immune thrombocytopenia (ITP) whose condition did not improve with standard oral treatments. Participants receive weekly injections of romiplostim and four weekly infusions of …

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 tests an experimental drug called Anti-CD38 antibody in 60 children aged 6 and older who have immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. The participants have not responded well to standard treatments.…

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 early-stage study tests a new drug called PRG-1801 in 6 adults with immune thrombocytopenia (ITP) that hasn't improved with standard treatments. The goal is to see if it is safe and can raise platelet counts to reduce bleeding risk. Participants receive a single infusion and…

This phase 2 trial tests two doses of peginterferon alfa-2b in 27 adults with essential thrombocythemia, a blood disorder causing too many platelets, who did not respond to or could not tolerate standard therapy. Participants receive weekly injections for up to 96 weeks. The stud…

This study tests a drug called herombopag in people with chronic liver disease who have low platelet counts and need surgery. The goal is to see if the drug can safely raise platelet levels enough to reduce bleeding risk during the procedure. About 101 adults with mild to moderat…

This phase 2 trial tests a two-step, lower-intensity transplant method for people with various blood cancers. The goal is to see if this gentler approach reduces the chance of dying from the transplant itself. About 63 participants will receive a combination of radiation, chemoth…

This study tests whether giving a whole blood transfusion can help children under 5 with severe malaria and very low platelet counts survive. The trial is enrolling 132 children in Zambia. Researchers will compare death rates and blood changes between those who get a transfusion …

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 early-phase trial tests a new drug called OM336 in 32 adults with autoimmune cytopenias, conditions where the immune system attacks blood cells. The study focuses on safety and how the drug moves through the body, not yet on curing the disease. Participants must have tried a…

This early-stage trial tests a treatment called CAR-T cell therapy for people with severe autoimmune diseases that haven't responded to other treatments. The therapy uses a patient's own immune cells, modified to target and reset the immune system. Only 3 participants will be enr…

This early-phase study is testing a new drug called CC312 in 18 adults with severe autoimmune diseases like lupus and rheumatoid arthritis that haven't improved with standard treatments. CC312 is a type of immune therapy designed to target and destroy faulty B cells, similar to C…

This early-phase study tests a new cell therapy called BZE2204 in 20 people with lupus, myositis, or immune thrombocytopenia (ITP) that has not responded to standard treatments. The therapy uses a patient's own immune cells, modified to target and calm the overactive immune syste…

This early-stage trial is testing a single infusion of cells from umbilical cord blood (called XJ-MK-002) in 16 cancer patients whose platelet counts have dropped due to chemotherapy or other cancer treatments. The main goal is to see if the treatment is safe and tolerable, and t…

This study tests whether adding teriflunomide (a drug used for multiple sclerosis) to standard high-dose dexamethasone works better than dexamethasone alone for adults newly diagnosed with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, caus…

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 aims to develop a non-invasive method to detect and track fibrosis (scarring) in myelofibrosis, a serious blood disorder. Currently, doctors must perform a bone marrow biopsy to check fibrosis. The researchers will test whether a blood test and a special PET scan can i…

This study tests whether tranexamic acid (TXA), a drug that prevents blood clots from breaking down, can safely reduce bleeding in people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). About 75 outpatients receiving less-intensive chemotherapy will be rando…

This phase 2 trial tests whether a curcumin supplement (C3 complex with piperine) can reduce inflammation and improve symptoms in people with certain blood disorders, including CCUS, low-risk MDS, and MPNs. Thirty participants will receive either curcumin or a placebo for 12 mont…

This study tests N-Acetylcysteine (N-AC), an antioxidant, in 27 adults with myeloproliferative neoplasms (MPN) like essential thrombocythemia, polycythemia vera, or myelofibrosis. The goal is to find the best dose that safely reduces symptom burden, measured by a symptom score. P…

This study tests a drug called QL0911 for people whose platelet levels drop too low due to cancer treatment. Low platelets can delay chemotherapy and cause bleeding risks. The trial will compare QL0911 to a placebo in 235 adults aged 18-75 to see if it safely raises platelet coun…

This phase 2 trial is testing whether the drug ruxolitinib (Jakafi) can reduce symptoms like fatigue and itching in people with low-risk essential thrombocythemia (ET) or polycythemia vera (PV). About 60 participants will take the pill daily, and researchers will measure symptom …

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 pilot study tests whether a 12-week nutrition and exercise program is feasible for patients with myeloproliferative neoplasms (MPNs), a group of blood cancers. Thirty participants will take virtual and in-person classes to learn healthier eating and physical activity habits.…

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 aims to learn more about Hermansky-Pudlak Syndrome (HPS), a rare inherited disease that causes light skin/eye color, bleeding problems, and often deadly lung scarring. Researchers will follow 600 people with HPS and their family members to track how the disease progres…

This study, called CEPEC, is a large platform trial enrolling 5,500 ICU patients across multiple countries. It tests different approaches to three common ICU treatments: how low to let blood pressure go with vasopressors, when to give platelet transfusions, and how to provide nut…

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

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 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 natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study looks at whether regular cannabis users can adjust how much they vape when using high-potency concentrates (30%, 60%, and 90% THC). Researchers will measure blood THC levels, puffing behavior, and subjective feelings of intoxication. The goal is to understand if people…

This study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

This study is testing whether a specialized blood test (MAIPA) can accurately identify autoimmune thrombocytopenia (ITP) in patients with low platelet counts. Currently, ITP is diagnosed by ruling out other causes, which can be slow and uncertain. Researchers will collect blood s…

This study aims to learn more about Chediak-Higashi syndrome, a rare genetic disorder that causes light skin and hair, easy bruising, and frequent infections. Researchers will observe up to 60 patients over time, collecting clinical and genetic data to better understand the disea…

This study is testing whether a fast blood test called ROTEM can accurately estimate platelet counts in pregnant women who have low platelets or severe bleeding after delivery. The goal is to help doctors make quicker, safer decisions about anesthesia and blood transfusions. Abou…

This early-stage study tests a new drug called ZE74-0282 in 40 healthy adults aged 18-55. The main goal is to check its safety and how the body handles it, not to treat any disease. Participants receive either the drug or a placebo, and researchers monitor side effects and drug l…

This study looks at how a natural citrus compound called d-limonene affects the body's response to THC, the main psychoactive ingredient in cannabis. Researchers will give healthy adults oral doses of THC, d-limonene, or both, and measure their feelings of drug effect and liking.…

This study is testing a new technique called optical genome mapping (OGM) to find genetic changes in blood cancers like polycythemia vera, essential thrombocythemia, and myelofibrosis. Researchers will compare OGM with standard genetic tests in 300 patients who need a bone marrow…

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 looks at how the immune system reacts to myeloproliferative neoplasms (MPN), a group of blood cancers. Researchers will collect blood and bone marrow samples from 150 adults with or without MPN to find special immune cells (T cells) that recognize and attack MPN cells.…

This study looks at blood samples from 44 people with thrombotic thrombocytopenic purpura (TTP) to understand how certain immune cells (B and T lymphocytes) react to the ADAMTS13 protein during different stages of the disease. The goal is to find early warning signs that could pr…

This study creates a nationwide registry and biobank for children diagnosed with myeloproliferative neoplasms (MPNs)—rare blood cancers that cause overproduction of blood cells. By collecting medical data and blood samples from up to 500 children across Germany, researchers hope …

This study looks at how CBD and THC, two compounds found in cannabis, affect inflammation and brain health in people with HIV. About 90 participants will take both substances in a random order over six weeks. Researchers will measure blood markers and brain function to better und…

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 back at medical records of 4 severely injured trauma patients who needed large blood transfusions. It compares two drugs that help blood clot—prothrombin complex concentrate and recombinant activated factor VIIa—to see which causes fewer dangerous blood clots and…

This study aims to understand exactly how the drug interferon attacks cancer stem cells in people with myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will analyze blood samples from 80 adults to see why some patients respond well while others do not. T…

This research study collects blood, bone marrow, and tissue samples from people with cancer, blood disorders, and healthy volunteers. The goal is to study how the immune system changes during different treatments, including immunotherapy and chemotherapy. By comparing samples fro…

This study looks at whether a specific gene, TNFAIP3, is linked to immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare blood clotting disorder. Researchers will compare genetic data from 400 people with iTTP and healthy volunteers to see if gene variations affect d…

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 is looking for the genetic mutation that causes polycythemia (too many red blood cells) and thrombocytosis (too many platelets). Researchers will analyze blood samples from 200 participants to identify the faulty gene. Finding this gene could help design new treatments…

This study is for adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis who are starting their first treatment. Researchers want to find out why some patients respond well to standard drugs while others do not. They will look at genetic and immune system …

This study looks at whether changes in the level of a specific gene mutation (CALR) in the blood can help predict when certain blood cancers (myeloproliferative neoplasms) get worse. Researchers will collect blood samples from 260 adults diagnosed between 2006 and 2020 and track …

This study follows 500 children aged 6-17 diagnosed with immune thrombocytopenia (ITP), a rare autoimmune disease that causes low platelet counts and bleeding. Researchers will collect data on symptoms, treatments, and outcomes over several years to build a model that predicts ho…

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study looks for substances in the blood that might be linked to fatigue in people with low platelet counts (thrombocytopenia). Researchers think a protein called BDNF, which is stored in platelets, could play a role. They will measure BDNF levels in 280 patients and healthy …

This long-term observational study is enrolling 5,000 adults with myeloproliferative neoplasms (MPNs)—rare blood cancers like polycythemia vera and myelofibrosis. Researchers will collect health records, patient surveys, and insurance data over at least five years to track sympto…

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 looking for better ways to predict blood clots in people with myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will measure specific markers in the blood of 300 participants to see if they can tell who is at higher risk for clots. The goal …

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 whether platelets—small blood cells that help with clotting—are found inside brain and spinal cord tumors. Researchers want to see if the presence of platelets in these tumors is linked to how long patients survive. The study will involve 50 adults who are hav…

This study looks at what happens when people who regularly use both marijuana and tobacco use them together. Researchers will measure how much THC and nicotine get into the blood and how the body responds. The goal is to better understand the health effects of using both substanc…

This study is a national registry in France that collects information on adults newly diagnosed with immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA). It aims to describe how these diseases progress and how well treatments work in real life. Researchers will tr…

This study examines why some patients with essential thrombocythemia (ET) later develop polycythemia vera (PV), a related blood condition. Researchers will review medical records of 678 adults to identify risk factors for this change and compare outcomes between those who progres…

This study is testing whether different doses of cannabidiol (CBD) can reduce the psychoactive effects of THC in healthy adults who use cannabis occasionally. One hundred participants will inhale various combinations of CBD and THC and report their experiences. The goal is to fin…

This study looks at the best way to treat blood clots in cancer patients who also have low platelet counts, which raises bleeding risk. Researchers will compare two approaches: full-dose blood thinners with platelet transfusions versus a reduced dose without transfusions. The goa…

This study looks at a type of immune cell called monocytes in people with certain blood cancers (polycythemia vera, essential thrombocythemia, and myelofibrosis). Researchers will analyze blood samples from 70 newly diagnosed patients to find unique patterns in these cells. The g…

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 collects blood, tissue, and body fluid samples from up to 15,000 people with blood cancers or related disorders. The samples are stored in a central lab for future research on diagnosis, prognosis, and immune monitoring. Participants do not receive any treatment as par…

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 looks at how THC, the active ingredient in cannabis, temporarily affects memory, learning, and balance in adults aged 21 to 55 who have used cannabis before. Ten participants will complete memory tasks and balance tests inside an fMRI scanner after taking either THC or…

This study reviews medical records from over 1,200 people with immune thrombotic thrombocytopenic purpura (iTTP), a rare and life-threatening blood disorder. The goal is to understand long-term health problems, such as heart disease and thinking issues, that survivors face. By an…

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 aims to uncover inherited (germline) genetic factors that make some people more likely to develop myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will analyze DNA from 313 patients diagnosed at a young age or with a family history of blood ca…

This study follows 100 people with confirmed dengue to identify which symptoms and lab results predict hospitalization. It also tracks how often blood transfusions are given and their outcomes. The goal is to improve care for dengue patients, not to test a new treatment.

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…

This study is collecting blood, urine, and stool samples from 500 children with autoimmune diseases and healthy controls. Researchers will analyze these samples to find molecular patterns that could help diagnose these conditions and guide treatment decisions. The goal is to crea…