Blood platelet disease

This study looks at 112 people with immune thrombocytopenia (ITP) whose platelet counts didn't improve after standard treatment with rhTPO. After a two-week break, they will either get a higher dose of rhTPO or switch to a different drug called eltrombopag. The goal is to see whi…

This trial investigates whether combining atorvastatin (a cholesterol-lowering drug), N-acetyl-L-cysteine (an antioxidant), and romiplostim (a platelet-boosting drug) can help adults with immune thrombocytopenia (ITP) who no longer respond to steroids. The goal is to see if this …

This phase 2 trial tests whether adding baricitinib to standard TPO-RA therapy helps adults with immune thrombocytopenia (ITP) who did not respond to TPO-RA alone. One hundred participants will be randomly assigned to receive either baricitinib plus TPO-RA or TPO-RA alone for 14 …

This phase 2 trial tests whether adding ianalumab to eltrombopag helps children aged 5 to 17 with primary immune thrombocytopenia (ITP) who didn't improve after steroids. The goal is to see if the combination can raise platelet counts and prevent bleeding. About 36 children will …

This study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…

This phase 2 study tests whether the drug hetrombopag can prevent dangerously low platelet counts caused by chemotherapy in breast cancer patients at high risk. About 126 participants will be split into three groups: one getting no preventive drug, one taking hetrombopag continuo…

This study looks at whether a higher dose of the blood thinner clopidogrel works better for people with essential thrombocythemia (ET), a rare blood disorder that raises the risk of dangerous blood clots. The standard dose may not work well for many ET patients, so researchers wi…

This study tests a new drug, INCA033989, against the best current treatments for people with a rare blood cancer called essential thrombocythemia who have a specific gene mutation (CALR). About 426 adults who have already tried at least one prior therapy will be randomly assigned…

This study tests a pill called selinexor in 15 adults with chronic blood cancers (MPN) whose blood cell counts are too high. The goal is to see if selinexor can safely bring those counts down and ease symptoms. Participants take the drug once a week for up to 3 months and are fol…

This early-stage study tests a new drug called JCXH-213 in adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial includes only 4 people whose ITP did not improve with standard treatments. The main goal is to…

This early-phase trial tests an injection called KSVCBD, a type of CAR-T cell therapy, in 60 adults with severe autoimmune diseases like lupus and scleroderma. The goal is to see if it's safe and to get a first look at whether it might help control the disease. Participants recei…

This phase 2 trial tests a personalized CAR T cell therapy called zolacabtagene autoleucel in 52 adults with chronic immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA). The treatment uses the patient's own immune cells, modified to target and destroy faulty B cel…

This early-phase study tests whether combining two drugs (Zuberitamab and Orelabrutinib) can help people with immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA) that hasn't responded to other treatments. The drugs work together to calm the immune system and stop …

This study tests all-trans retinoic acid (ATRA), a form of vitamin A, in adults with immune thrombocytopenia (ITP) whose platelet counts stay low despite steroid treatment. About 192 participants will receive either ATRA or a placebo for 24 weeks. The goal is to see if ATRA can s…

This early-stage trial tests a new therapy called universal STAR-T cells in about 6 adults with autoimmune thrombocytopenia (low platelets) that hasn't responded to standard treatments. The cells are designed to target and calm the overactive immune system. The main goals are to …

This early-phase study tests a new treatment called IASO206 for people whose autoimmune hemolytic anemia (AIHA) has not responded to standard therapies. AIHA is a condition where the immune system mistakenly destroys red blood cells, causing fatigue and other symptoms. The study …

This phase 2 trial tests ropeginterferon alfa-2b, a long-acting immune protein, in 35 adults with rare blood cancers like CMML and MDS/MPN overlap syndromes. The drug aims to slow cancer cell growth by boosting the immune system. Researchers will check safety, best dose, and how …

This study tests the drug baricitinib in 28 adults who have dangerously low platelet counts after a stem cell transplant and did not improve with standard treatments. The goal is to see if baricitinib can safely raise platelet levels enough to reduce the need for transfusions. Th…

This early-stage trial tests UX016, a drug designed to help people with GNE myopathy, a rare genetic muscle-weakening disease. Researchers will give the drug or a placebo to 24 adults to see if it is safe and improves muscle strength. The study is not yet recruiting.

This phase II trial tests two oral drugs, selinexor and pacritinib, in 26 adults with myelofibrosis who have anemia and low platelets and have not taken JAK inhibitors before. The main goal is to see if the combination can shrink the spleen by at least 35%. The study also tracks …

This early-phase trial tests a new drug called TGD001 in about 70 adults with acute episodes of immune-mediated thrombotic thrombocytopenic purpura (iTTP) or related blood clotting conditions. The main goal is to check the drug's safety and how the body processes it, while also l…

This phase 3 trial tests QL0911, a weekly injection, in 60 children aged 1 to 17 with chronic immune thrombocytopenia (ITP) who have low platelet counts. The goal is to see if the drug can safely raise platelet levels to reduce bleeding risk. Participants receive either QL0911 or…

This early-stage trial tests a new cell therapy called YTS109 for people with autoimmune hemolytic anemia that hasn't improved after at least three prior treatments. The therapy uses specially engineered immune cells to target and destroy the faulty cells causing the anemia. The …

This early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…

This early-phase trial will test a new type of CAR T-cell therapy, called QT-019C, in 27 adults with refractory immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The therapy uses donor-derived immune cells engineered to targe…

This phase 2 study tests whether combining orelabrutinib (a targeted drug) with romiplostim (a platelet booster) can improve platelet counts in adults with chronic immune thrombocytopenia (ITP) who haven't responded well to standard treatments. About 28 participants will receive …

This early-phase study tests a new mRNA therapy that targets CD19, a protein on immune cells, to treat autoimmune diseases like lupus and immune anemia. The treatment uses lipid nanoparticles to deliver mRNA that helps the body clear harmful B cells. About 47 participants aged 14…

This Phase 2 trial tests whether combining two platelet-boosting drugs (hetrombopag and rhTPO) works better than hetrombopag alone for cancer patients with dangerously low platelets from treatment. About 204 adults with solid tumors will be randomly assigned to one of the two gro…

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

This early-stage trial is testing three different daily doses of zanubrutinib (80, 160, or 240 mg) combined with high-dose dexamethasone in 60 adults newly diagnosed with immune thrombocytopenia (ITP). The goal is to find the safest and most effective dose that helps maintain pla…

This early study tests a new treatment called CD19 UCAR-T cells for people with autoimmune diseases like lupus, vasculitis, and Sjögren's syndrome. The therapy uses modified immune cells to target and calm the overactive immune system. The main goals are to check safety and see i…

This early-phase trial tests a new cell therapy called YTS109 in 13 adults with autoimmune hemolytic anemia that has not responded to at least three prior treatments. The therapy uses specially engineered immune cells to target and control the disease. The main goals are to check…

This study tests whether adding all-trans retinoic acid (ATRA) to the drug avatrombopag can help adults with immune thrombocytopenia (ITP) maintain normal platelet levels without needing ongoing treatment. About 248 participants who have not responded well to first-line therapy o…

This study tests a drug called hetrombopag to see if it can prevent or reduce low platelet counts caused by antibody-drug conjugate (ADC) chemotherapy in breast cancer patients. The trial will include 72 adults aged 18-75. Researchers will measure how many patients develop severe…

This study tests a drug called romiplostim, given as a weekly shot, in 100 children aged 1 to 18 with chronic immune thrombocytopenia (ITP) who haven't been helped by standard treatments. The goal is to see if it can safely raise platelet counts and reduce bleeding. Children will…

This study tests whether a short course of the antibiotic rifaximin can improve how well the blood thinner ticagrelor works in people who have had a heart attack. The idea is that unhealthy gut bacteria may interfere with the drug, and clearing them out could help prevent future …

This study tests whether herombopag tablets can prevent low platelet counts in 45 women with HER2-positive breast cancer who are receiving T-DM1 therapy. T-DM1 is effective but often causes low platelets, especially in Asian patients, which can force treatment delays or dose redu…

This early-stage trial tests golidocitinib, an oral drug, in adults with immune thrombocytopenia (ITP) whose platelet counts remain low despite prior treatments. The study has two parts: first, finding a safe dose, then comparing the drug against a placebo in a blinded fashion. T…

This study tests a three-step treatment for adults with severe immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. Participants first get steroids and a platelet-boosting protein in the hospital, then switch to an oral drug (el…

This early-stage trial tests a new CAR-T cell therapy called inaticabtagene autoleucel for people with autoimmune hemolytic anemia (AIHA) that hasn't improved after at least three prior treatments. The study will enroll 12 adults and primarily check for safety and the right dose.…

This study tests a drug called Romiplostim N01 in 106 cancer patients whose platelets drop due to chemo, radiation, or immunotherapy. The goal is to see if it can safely raise platelet counts better than the current standard treatment. Patients will be randomly assigned to receiv…

This phase 3 trial will compare two second-line treatments for immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risk. About 220 adults who didn't respond to steroids will be randomly assigned to take avatrombopag (a daily pil…

This phase 2 trial tests linprixel, a daily pill, in 22 adults with relapsed or refractory autoimmune hemolytic anemia (AIHA). The study aims to see if the drug can improve red blood cell counts after 12 weeks. Participants start at 40mg daily, with possible dose increases if nee…

This study tests whether a 12-week personalized exercise program can reduce fatigue and improve physical function in people with myeloproliferative neoplasms (MPN) and chronic myeloid leukemia (CML). Participants will do supervised aerobic and resistance training twice a week plu…

This study tests a digital app called WELL-MPN designed to help adults with myeloproliferative neoplasms (MPNs) improve their diet, physical activity, symptoms, and emotional well-being. About 75 participants will be randomly assigned to use the app or receive standard written li…

This study investigates how platelet transfusions affect the function of small blood vessels and inflammation in intensive care patients with low platelet counts. Researchers measure blood vessel response using a laser device on the skin and track levels of various biomarkers in …

This study looks at children with immune thrombocytopenic purpura (ITP) who also have certain antibodies, as they are at higher risk of developing lupus. Researchers will analyze blood samples to track immune cell changes and markers that may signal the transition to lupus. The g…

This study tests whether two different capsule forms of the experimental drug SKI-O-703 are absorbed similarly in the body. About 48 healthy adults (Korean and Caucasian) will take a single dose of each form on separate days, with blood samples taken to compare drug levels. The r…

This study looks at how well platelets work in patients on a life-support machine called ECMO, which helps people with severe lung or heart problems. The goal is to understand why these patients often have dangerous bleeding. Researchers will measure platelet function over time a…

This study aims to create a score that uses a special blood test to predict bleeding risk in people with blood cancers and very low platelet counts. Researchers will take an extra blood sample from 410 participants and analyze it with viscoelastometric tests. The goal is to ident…

This study looks at how eltrombopag is actually used and how well it works in patients with immune thrombocytopenia (ITP) who have already tried steroids. Researchers will collect data from healthcare providers in the US, UK, and Germany, focusing on platelet count improvements w…

This study looks at whether a new test called T-TAS can help predict bleeding after heart surgery. Researchers will take blood samples from 30 adults before, right after, and 24 hours after surgery. They will compare T-TAS results with standard tests and track how much bleeding a…

This study looks back at medical records of people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder that causes dangerous blood clots. Participants received a manufactured version of the missing ADAMTS13 enzyme through an early access pr…

This study is creating an AI tool to help doctors decide the best heart treatment for people who have both immune thrombocytopenia (ITP) and coronary artery disease. Researchers will look at past patient data to build the tool, then test it on 600 new patients. The goal is to bet…

This study looks at whether young people (ages 8–25) with chronic immune thrombocytopenic purpura (ITP) have lower aerobic fitness than healthy peers. Fatigue is a major problem for many with ITP, and the researchers want to see if it's linked to a 'vicious cycle' of being less a…

This study looks at two genes (IL-1β and IL-33) in 300 adults with low platelet counts caused by either primary immune thrombocytopenia (ITP) or lupus (SLE). Researchers will measure gene activity from a blood sample to see if these genes can help tell the two conditions apart. T…

This study will review medical records of 500 people with newly diagnosed or persistent immune thrombocytopenia (ITP) across multiple hospitals in China. Researchers will look at what treatments patients actually received and how well they worked, without influencing their care. …

This study will look at 100 adults with low platelet counts to see how many have an enlarged spleen and whether that affects their symptoms, complications, or response to treatment. The goal is to better understand the role of the spleen in these blood disorders. No new treatment…

This study will measure vitamin D levels in 84 adults newly diagnosed with immune thrombocytopenia (ITP), a bleeding disorder. Researchers want to see if vitamin D levels are linked to how well patients respond to first-line steroid therapy. The goal is to gather knowledge, not t…