Thrombotic microangiopathy

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 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 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 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 Phase II trial tests a drug called NM8074 in 12 adults with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots, low platelets, and kidney injury. The drug is given by IV infusion over several weeks. The goal is to see if NM8074 can bring bloo…

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 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 study will follow about 80 Polish patients with atypical hemolytic uremic syndrome (aHUS) who are receiving ravulizumab (Ultomiris) as part of routine care. Researchers will collect data from medical records and follow patients over time to see how well the drug controls the…

This study tests a drug called iptacopan in 30 people who have a serious blood vessel problem after a stem cell transplant. The drug aims to block a part of the immune system that damages blood vessels. Researchers will check if it helps patients live longer and recover organ fun…

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 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 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 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 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 study tests whether taking ginger powder for the first three days of each period can reduce heavy menstrual bleeding in women with coagulation disorders (bleeding problems). 74 women will be randomly assigned to receive either ginger or a placebo for six cycles. The goal is …

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 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 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 will follow 85 adults receiving stem cell transplants to track a serious but often missed complication called TA-TMA. Researchers want to find out how common it is and how it affects survival. Participants will provide blood samples and have standard follow-up care.

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 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…