Primary thrombocytopenia

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 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 early-phase study tests whether combining mesenchymal stem cells (MSCs) with specially engineered immune cells (CAR-T cells) is safe and can help control autoimmune diseases. About 30 adults with established autoimmune conditions will receive the treatment and be monitored f…

This study tests whether specially engineered immune cells, called CAR-T cells, can safely treat autoimmune diseases. The cells are designed to target and suppress B cells, which play a key role in many autoimmune conditions. The trial involves 30 adults and focuses on safety and…

This study is testing a new blood filter called the Plasma Filter PX2. It is used in a procedure called therapeutic plasma exchange, which removes harmful substances from the blood of people with autoimmune diseases. About 46 adults will take part, and the study will check if the…

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 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 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 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 bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

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 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 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 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 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-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 early-stage study tests a new mRNA-based treatment (ABO2203) for people with autoimmune diseases that haven't improved with standard care. The therapy works by directing the body's immune cells to attack and control overactive immune cells causing the disease. The study will…

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 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 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 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 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-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 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 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 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 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 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-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-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 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 whether the shingles vaccine (RZV) works and is safe for adults with autoimmune rheumatic diseases like rheumatoid arthritis or lupus who are taking immune-suppressing drugs. Two hundred participants will receive two doses of the vaccine. Researchers will me…

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 is looking for the genes that cause certain immune system problems. Researchers want to understand why some people get sick and others don't, even in the same family. Up to 5,000 people with immune disorders and their relatives will give blood or cheek cell samples for…

This study will analyze DNA from up to 20,000 people, including those with immune disorders and their relatives, to find genetic variants linked to conditions like atopy, autoimmunity, and primary immunodeficiency. Researchers will also study how best to return genetic results to…

This study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…

This study is measuring a protein called STIM1 on the surface of immune cells in 670 people with various autoimmune diseases. The goal is to find which diseases have high levels of this protein, which could help researchers develop new antibody-based treatments. Participants prov…

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 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 collects and freezes testicular tissue from boys and young men who are at risk of becoming infertile due to cancer, autoimmune disorders, or their treatments. The tissue is stored in a research bank, with the hope that future medical advances will allow it to be used 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 is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

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 looks at 320 adults who are about to start strong immune-suppressing therapy and have already had at least two COVID-19 shots. Researchers want to find out if giving a COVID-19 booster right before starting therapy or waiting six months provides better protection. Part…

This study is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…

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 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 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 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 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 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…