Blood coagulation disease

This early-stage study tests a new drug called SR604 in healthy people and those with hemophilia A, B, or factor VII deficiency. The main goal is to check safety and how the drug moves through the body. About 31 participants will receive single or multiple doses to find the right…

This study looks at whether Emicizumab, a newer drug, prevents bleeding during sports better than standard Factor VIII in children with moderate to severe hemophilia A. About 72 kids aged 6 to 19 who already play sports and take one of these drugs will be followed. The goal is to…

This early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…

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 looks at the long-term safety and effectiveness of a medicine called marstacimab for people with severe hemophilia A or B. Participants receive regular injections to help prevent bleeding episodes. The goal is to see if this treatment is safe and reduces bleeds over ti…

This study tests a new medicine, HMB-002, in about 108 adults with von Willebrand disease (types 1 and 2). The goal is to check safety, how the drug moves in the body, and whether it helps control bleeding. It is the first time this drug is being tested in humans, starting with s…

This study tests Mim8, a drug that helps blood clot by mimicking a missing protein, in people with hemophilia A (with or without inhibitors). Over 450 participants will receive injections for up to 5.5 years to see if it safely prevents bleeding episodes. The goal is to provide a…

This study follows 250 people with hemophilia A who are taking efanesoctocog alfa to prevent bleeding. Researchers want to see how well it protects joints from bleeds over time in everyday life. Participants will be observed for several years to track bleeding rates and treatment…

This study gives people with moderate or severe hemophilia A who finished a previous trial continued access to the drug efanesoctocog alfa. The drug is a lab-made clotting factor that helps prevent bleeding episodes. Researchers will monitor safety and how well it controls bleedi…

This phase 3 trial is testing a drug called fitusiran in about 85 boys aged 1 to under 12 with severe hemophilia A or B, including those with inhibitors that make standard treatments less effective. The drug is given as a regular shot under the skin to help prevent bleeding episo…

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 phase 3 trial tests a new drug called FRSW107, a lab-made clotting factor VIII fused with a protein that helps it last longer in the body. The study enrolls males aged 12 to 65 with severe hemophilia A who have had at least 150 prior exposure days to factor VIII products. Pa…

This early-stage trial tests a gene therapy for people with hemophilia A, a bleeding disorder. The treatment uses a modified virus to deliver a working gene into the patient's own stem cells, which are then infused back. The goal is to help the body produce clotting factor VIII, …

This early study tests a new drug called BAY 3389934 in 36 hospital ICU patients with sepsis-induced coagulopathy, a condition where the body forms dangerous blood clots. The main goal is to check safety and find the right dose. Researchers will monitor side effects and blood clo…

This study tests a new gene therapy called SPK-8011QQ in 5 adult men with severe or moderately severe hemophilia A. The goal is to see if it is safe and tolerable. Participants receive a one-time intravenous dose, and researchers monitor for side effects and lab changes.

This trial compares two blood products—prothrombin complex concentrate (PCC) and fresh frozen plasma (FFP)—for controlling bleeding in people undergoing heart valve surgery. Doctors use a special test to guide which product to give and measure how quickly bleeding stops. The goal…

This Phase 3 trial tests TAK-330, a drug designed to reverse the effects of common blood thinners (Factor Xa inhibitors) in patients who need urgent surgery. About 440 adults will be randomly assigned to receive TAK-330 or standard care before their procedure. The main goal is to…

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 study tests a medicine called emicizumab to prevent bleeding episodes in people with Type 3 von Willebrand disease, a severe inherited bleeding disorder. About 75 participants, from infants to adults, will receive either emicizumab or continue their current treatment. The go…

This study tests a single-dose gene therapy called CSL222 (Etranacogene dezaparvovec) in 35 adults with severe or moderately severe hemophilia B who have detectable AAV5 antibodies—a group often excluded from gene therapy. The goal is to see if it reduces bleeding episodes and is…

This program provides concizumab, a daily injection under the skin, for free to people with congenital hemophilia who need better treatment. Participants will be monitored for safety and how well the medicine works over several years. The program is for those with severe hemophil…

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 Phase 2 trial tests an injection called SR604 in 24 adults with von Willebrand disease, a bleeding disorder. The goal is to see if it safely reduces the number of bleeding episodes. Participants receive multiple doses, and researchers track bleeding rates and quality of life…

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 tests an experimental injection called SR604 in 76 adults with hemophilia A, B, or congenital factor VII deficiency. The goal is to see if it is safe and can reduce bleeding episodes. Participants will receive either a single dose or multiple doses, and researchers wil…

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 Phase 3 trial tests a new drug called NXT007 (Zemocimig) against standard Factor VIII treatment to prevent bleeding in people with severe or moderate hemophilia A who do not have inhibitors. About 126 participants aged 12 and older will receive either NXT007 as a shot under …

This study compares a new drug, NXT007, to an existing one (emicizumab) for preventing bleeding episodes in people aged 12 and older with hemophilia A. Participants may have severe, moderate, or mild disease, with or without factor VIII inhibitors. The goal is to see if NXT007 is…

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement th…

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 new drug, NXT007, in people with severe or moderate hemophilia A. The drug is given as a shot under the skin, first every two weeks, then every four weeks. The goal is to see if it is safe and helps control bleeding. About 60 adults, teens, and children will ta…

This study looks at the long-term safety and effectiveness of the drug iptacopan in people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). Participants must have completed a previous iptacopan study and still benefit from the drug. The goal is to moni…

This study looks at whether it is safe for males aged 12 and older with severe hemophilia A to switch from their current medicine (emicizumab) to a new one called fitusiran. About 20 participants will be followed for up to 28 months to check for side effects and how well the new …

This study tests a drug called Atenativ in people with a rare inherited condition that raises their risk of dangerous blood clots. The goal is to see if Atenativ can prevent clots during surgery or childbirth. About 38 adults and some teens will take part. The drug is given as an…

This early-stage study tests a new medicine called marstacimab in people with severe hemophilia A who want to switch from their current preventive treatment, emicizumab. The study includes 15 adolescents and adults who will receive weekly injections of marstacimab for 4 months. R…

This study tests a new drug called sutacimig in 18 adults with a rare inherited bleeding disorder (factor VII deficiency). The drug is given as a single shot under the skin to see if it is safe and can help prevent bleeding. The goal is to find a more convenient treatment option …

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 a drug called vonicog alfa to prevent bleeding in 24 children with severe von Willebrand disease, a condition that causes easy bruising and heavy bleeding. Participants receive the drug regularly for 12 months to see if it reduces bleeding episodes compared to th…

This study tracks 25 people in Japan with acquired hemophilia A who receive the drug OBIZER to control bleeding. Researchers will watch for side effects and check if the drug helps stop bleeding episodes. The study does not change how doctors treat patients—it simply collects rea…

This study tests an experimental gene insertion therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR/Cas9 to add a working copy of the clotting factor IX gene into the body's DNA, aiming to enable long-term production of…

This study is checking how safe OBIZUR is for adults with acquired hemophilia A, a rare condition that causes sudden and severe bleeding. Researchers will look at medical records and track side effects in about 9 people. The goal is to see how well the drug controls bleeding in e…

This study tests whether giving children with a severe E. coli infection extra fluids through an IV can prevent kidney failure. About 1,040 children aged 9 months to 21 years will take part. The goal is to see if this simple approach reduces the need for dialysis or long-term kid…

This study tests whether a step-by-step care plan (algorithm) can lower the chance of another serious stomach or nose bleed in people with von Willebrand disease. About 66 adults who have had such a bleed will be randomly assigned to either the new plan or standard care. The goal…

This study tests a drug called Atenativ, made from human plasma, in 120 adults who are resistant to heparin during planned heart surgery. Heparin is a blood thinner used to prevent clots during surgery, but some patients don't respond well. The drug aims to restore heparin's effe…

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 recombinant factor VIII product called Nuwiq can safely prevent bleeding during major surgery in people with severe hemophilia A who are already on emicizumab. About 28 male patients aged 12 and older will take part. The main goal is to see if the treat…

This study is testing a drug called Nuwiq, a lab-made clotting factor, to prevent excessive bleeding during major surgery in women and girls with hemophilia A. About 28 participants will receive Nuwiq around the time of their surgery. Researchers will check if the drug successful…

This study tests a new medicine called marstacimab in children aged 1 to 17 with severe hemophilia A or B. The medicine is given as a weekly shot under the skin to prevent bleeding episodes. Researchers will compare how well it works to the children's own past year of standard tr…

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

This study tests 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 phase 3 study is testing whether a low, personalized dose of FEIBA (aPCC) can safely control bleeding in children and adults with hemophilia A who have inhibitors and are already taking emicizumab. The study will measure how well the blood clots after FEIBA is given and trac…

This study is for adults with a rare blood disorder called aHUS who are currently receiving antibody infusions. Researchers want to see if switching to a daily pill called iptacopan is safe and works just as well to prevent dangerous blood clots. About 50 participants will take i…

This study tests whether giving a clotting protein called factor XIII early can reduce blood loss in women with heavy bleeding after childbirth. About 988 women having a vaginal delivery will be randomly assigned to receive either factor XIII plus standard care or standard care a…

This registry follows 100 adults and teens with hemophilia A or B who are prescribed marstacimab, a once-weekly shot under the skin. The main goals are to see if patients prefer this shot over IV treatments and to measure treatment burden after 6 months. Researchers will also tra…

This Phase 1 trial tests a gene therapy that uses a lentivirus to deliver a working clotting factor IX gene into patients' own stem cells. Ten males with severe hemophilia B will receive the modified cells. The goal is to see if it's safe and can raise clotting factor levels, pot…

This study tests an experimental oral medicine called Inno8 in 30 adults with hemophilia A. The goal is to see if it is safe and how the body processes it. Participants will take multiple doses over about 11 weeks. If successful, Inno8 could offer a more convenient way to control…

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 Phase 3 trial is testing STSP-0601, an injectable drug, to control bleeding episodes in people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The study will enroll 40 participants aged 12 to 70 and measure how well the drug s…

This Phase 3 study is testing a drug called KN057, given as a weekly injection under the skin, to prevent bleeding in people with hemophilia A or B (with or without inhibitors). The trial will enroll 70 males aged 12 to 65 and will monitor safety and how well the drug works over …

This study looks at whether a treatment called immune tolerance induction (ITI) can help people with haemophilia A who have developed inhibitors—antibodies that block standard clotting factor therapy. Researchers will give high doses of individualized factor VIII concentrates to …

This study is testing the drug ravulizumab in 20 people with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. The goal is to see if the drug improves platelet counts and kidney function over 26 weeks. All participants receive …

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 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 study tests whether emicizumab (Hemlibra), a medicine given as a shot under the skin, can safely prevent bleeding in people with severe von Willebrand disease (VWD) or combined VWD/hemophilia A. About 40 participants of any age (including infants) will receive the drug and b…

This phase 3 study tests a drug called VGA039, given as a shot under the skin, to prevent bleeding in people with Von Willebrand disease. The trial will include 60 patients aged 12 to 75 who have frequent bleeding episodes. Researchers will track how well the drug reduces bleeds …

This study tests whether the blood thinner apixaban can dissolve blood clots in the portal vein that develop more than a year after laparoscopic spleen surgery in people with cirrhosis. Twenty participants will take apixaban twice daily, and doctors will use ultrasound or CT scan…

This study looks at whether the blood thinner apixaban can safely dissolve blood clots in the portal vein (the main vein to the liver) that appear more than a year after spleen removal surgery. It involves 20 adults with cirrhosis who had their spleen removed. The goal is to see …

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 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 trial tests a one-time gene therapy called BBM-H803 for adults with severe hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor VIII. The study involves 55 participants and will check safety…

This study tests whether a concentrated clotting drug (fibrinogen) can better control major blood loss during surgery for pseudomyxoma peritonei, a rare abdominal cancer. About 90 adults will be randomly assigned to receive either the drug or a standard blood product during their…

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 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 is testing a drug called SCT800, a lab-made clotting factor, to prevent bleeding episodes in 36 boys with severe hemophilia A who have never been treated before. Participants receive regular infusions of SCT800, and researchers will monitor how many bleeding events occ…

This early-stage study tests a single injection of CITY-FXI, a drug designed to lower clotting risk by targeting a protein called Factor XI. It includes healthy adults and those with Factor V Leiden or prothrombin G20210A mutations, which increase clot risk. The main goal is to c…

This study in Japan looks at how safe and effective Vonvendi (rVWF) is for children under 18 with Von Willebrand Disease, a bleeding disorder. About 13 kids will receive the drug as part of their normal care for bleeding episodes or before surgery. Researchers will monitor side e…

This study follows 500 people with hemophilia B who received HEMGENIX gene therapy, plus a comparison group on standard factor IX treatment. Researchers will track bleeding rates and safety over several years to see if the gene therapy provides lasting control. The goal is to und…

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 study tests a medicine called prothrombin complex concentrate (PCC) to stop bleeding episodes in people with hemophilia A who have developed inhibitors (antibodies that block standard treatment). Thirty participants aged 12 to 65 will receive PCC on-demand during bleeding, w…

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 early-stage trial tests a single dose of a lab-made clotting protein (recombinant von Willebrand factor) in 12 adult patients on ECMO who have major bleeding. The goal is to see if the drug is safe and how it behaves in the body. If successful, it may lead to a new way to co…

This study compares two treatments to help blood clot in severely injured trauma patients who are bleeding heavily. One is a standard frozen blood product (cryoprecipitate) that takes time to thaw, and the other is a dry powder (fibrinogen concentrate) that can be given at the be…

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 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 study tests a new medicine called INM004 in 220 children with hemolytic uremic syndrome (HUS) caused by E. coli bacteria. The goal is to see if it helps the kidneys recover faster and reduces the need for dialysis. Participants are children aged 9 months to 18 years who have…

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 is testing the safety of a drug called SEVENFACT, a clotting factor made from the milk of genetically modified rabbits, in people aged 12 and older with hemophilia A or B who have inhibitors. The drug is already approved for treating bleeding episodes, and this trial w…

This study tests a drug called VONVENDI (recombinant von Willebrand factor) in 20 Chinese adults with von Willebrand disease, a bleeding disorder. The goal is to see if it is safe and helps control bleeding during surgery or dental procedures, sometimes combined with another drug…

This study looks at whether giving a medicine called Wilate to pregnant women with von Willebrand disease can prevent severe bleeding during and after childbirth. About 110 women will receive Wilate to keep their clotting factor levels high enough during delivery. The goal is to …

This study follows 300 teens and young adults (ages 10-24) with inherited bleeding disorders who choose either a levonorgestrel IUD or norethindrone acetate pills for heavy menstrual bleeding. Researchers will track bleeding patterns, satisfaction, and quality of life over six mo…

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 study tests a single-dose gene therapy called GS1191-0445 in 50 Chinese men with hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, helping the body produce its own clotting factor. The goal is to reduce bleeding episodes and the need f…

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 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 3 trial is testing a single dose of ANB-002, a gene therapy that delivers a working copy of the FIX gene, in 24 adults with hemophilia B. The goal is to see if it can reduce bleeding episodes compared to regular preventive factor IX infusions. Participants will be foll…

This study tests whether a once-weekly infusion of efanesoctocog alfa can improve joint swelling (synovial hypertrophy) in people with moderate to severe hemophilia A. Researchers will use ultrasound to measure changes in joint health over 12 months. The trial enrolls 35 particip…

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 study in Taiwan will follow about 100 people with moderate to severe hemophilia A who are already taking efanesoctocog alfa (a clotting factor) as part of their routine care. Researchers will track joint health, bleeding rates, and safety over up to 5 years using medical rec…

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 compares three different doses of the drug tranexamic acid in 150 adults having elective heart surgery with a heart-lung machine. The goal is to find which dose best controls bleeding and reduces the need for blood transfusions. Researchers will measure how long the dr…

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 program provides continued compassionate access to recombinant von Willebrand factor (rVWF) for children and adults with severe von Willebrand disease who completed a prior study and are benefiting from treatment. Participants must have no comparable alternative therapy avai…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

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 is testing a new blood test (the HMEC test) to see if it can better identify which patients with thrombotic microangiopathy (TMA) have a specific immune system problem driving their disease. TMA is a severe condition that damages kidneys and the brain. The researchers …

This study tests a small, battery-powered device called ClotChek™ that measures how fast a patient's blood clots using a fresh drop of blood. Researchers want to see if it can tell the difference between people on common blood thinners (rivaroxaban or apixaban) and those not on a…

This study tests whether a non-invasive light therapy called photobiomodulation can reduce pain and swelling in the knees of children with hemophilia who have joint bleeding. Twenty-four boys and girls aged 9 to 14 will receive the light treatment alongside standard care. Researc…

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 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 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 follows people of all ages who have or may have a RUNX1 gene variant, which can cause bleeding problems and increase the risk of blood cancers. Researchers will collect medical history, blood samples, and bone marrow over many years to understand how the condition deve…

This study looks at the tiny organisms living on our skin—like bacteria and fungi—and how they might differ in people with eczema (atopic dermatitis). Researchers will collect skin samples from healthy volunteers and patients aged 2 to 40 with moderate to severe eczema, as well a…

This study monitors the safety of Jivi (damoctocog alfa pegol) in 20 Korean patients with hemophilia A who have used other treatments before. Researchers will collect data from routine clinic visits over about 8 months per participant, looking for any medical problems. No extra t…

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 follows 2,000 children who receive stem cell transplants to better understand and detect lung injuries that can occur after the procedure. Researchers will track participants for two years, collecting samples and health data to find ways to diagnose problems earlier. T…

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 phase 1 trial at M.D. Anderson Cancer Center is testing whether it is safe to collect stem cells from people with RUNX1 familial platelet disorder, a rare inherited condition that raises the risk of bleeding and leukemia. Four participants will receive drugs to mobilize stem…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

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 collects blood, urine, bone marrow, and tumor samples from people with hairy cell leukemia and other blood cancers, as well as from healthy volunteers. Researchers will use these samples to study the diseases and develop new treatments called immunotoxins, which use an…

This study is for people with plasminogen deficiency (a rare blood disorder) and their close family members. Researchers will collect medical history and blood samples over several years to better understand how the disease develops and varies between people. No new treatments ar…

This study is observing how well three Factor VIII concentrates (Nuwiq, Octanate, Wilate) work in real-world settings for people with severe hemophilia A who have had little or no prior treatment. Researchers will track bleeding rates and side effects in about 200 participants of…

This study enrolls 100 adults who have or may have Factor V Leiden or Prothrombin G20210A mutations, which increase the risk of blood clots. Participants undergo genetic testing to confirm their status, then attend a site visit for basic health checks like blood pressure, ECG, an…

This study follows 173 people with hemophilia A or B who previously received a single-dose gene therapy in a Pfizer trial. Researchers will track bleeding episodes, clotting factor levels, and any long-term side effects like blood clots or liver issues. The goal is to understand …

This study uses brain MRI scans to look for tiny old bleeds (microbleeds) in 200 adults with severe hemophilia or von Willebrand disease. Researchers want to see how common these microbleeds are and whether they are linked to memory or thinking difficulties. Participants will als…

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 watches how blood clotting changes in 58 people after complex surgery to repair a thoraco-abdominal aortic aneurysm. Researchers will check for complications like kidney injury, heart problems, and clotting issues before and after surgery. The goal is to better underst…

This study is creating a risk assessment system to predict blood clotting abnormalities in women with polycystic ovary syndrome (PCOS). Researchers will analyze hormone, metabolic, and genetic factors in 100 participants aged 14 to 45. The goal is to help doctors personalize care…

This study looks at whether ultrasound and other imaging can find hidden joint changes in people with hemophilia who have no obvious symptoms. Researchers will follow 46 patients for up to 12 weeks, using scans and physical exams to see if these hidden changes are active or inact…

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 is checking if a computer model can accurately predict why some people have a prolonged blood clotting time (APTT). The model uses data from routine blood tests to tell if the cause is harmless or a sign of a clotting disorder. Researchers will test it on 1,500 patient…

This study is for people aged 16 to 70 with von Willebrand disease (VWD), a bleeding disorder. Researchers will collect information on bleeding events, treatments, and quality of life over time. The goal is to understand the real-world impact of VWD and use this data as a baselin…

This study creates a web-based registry for people with severe von Willebrand disease (sVWD), a rare bleeding disorder. Researchers will collect information from up to 400 participants about their medical history, symptoms, and lab results. The goal is to build a detailed databas…

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 reviews medical records of 200 people diagnosed with a rare blood and kidney disease called aHUS in Egyptian hospitals. Researchers want to learn about patient symptoms, treatments, and long-term health to help doctors diagnose and manage the disease better. No new tre…

This study looks at cancer patients who have a type of blood clot called superficial venous thrombosis (SVT). About 15-20% of SVTs happen in people with cancer, but there are no clear treatment guidelines for them. Researchers will observe 1,576 patients to see how different bloo…

This study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This study looks at 36 adults with severe hemophilia A to understand how joint health, pain, balance, and falls impact their quality of life. Researchers will measure joint damage, balance using a special platform, pain levels, and fall history. The goal is to find which factors …

This study is collecting blood and other samples from 5,000 children and young adults (under 21) who are already taking certain medications as part of their regular care. The goal is to understand how these drugs move through and affect children's bodies, so doctors can prescribe…

This study is testing whether patients recovering from hip or knee replacement can safely skip elastic compression stockings and rely only on blood-thinning drugs to prevent dangerous blood clots. Over 1,200 participants will be randomly assigned to receive either drugs plus stoc…

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 well hip, knee, shoulder, and other joint replacement surgeries work in adults with inherited bleeding disorders like hemophilia. Researchers will collect information from medical records and follow patients over time to understand risks, complications, an…

This study aims to create a reliable lab test to monitor how well anti-TFPI antibodies work in people with severe hemophilia. Researchers will take a single blood sample from 11 patients already on anti-TFPI therapy and test it in the lab. The goal is to better predict and manage…

This study aims to improve diagnosis for people with rare genetic diseases whose standard genetic tests came back negative. Researchers will take a small skin sample and analyze RNA to find hidden genetic changes. The study involves 105 participants, including healthy volunteers …

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 why people with hemophilia (a bleeding disorder) are less likely to die from heart disease. Researchers will use a new, detailed CT scanner to compare heart artery health in 80 men aged 45+ with hemophilia to similar men without it. The goal is to understand h…

This study collects DNA from 1200 patients receiving routine care at the University of Chicago. Researchers look for genetic differences that might affect how well a drug works or how likely it is to cause side effects. The goal is to see if giving this genetic information to doc…

This study looks at how four types of central venous catheters (thin tubes placed in large veins) affect the body's clotting system. Researchers will take blood samples from 88 adults right after catheter insertion and again after flushing, to measure clotting changes. The goal i…

This study follows 500 mother-child pairs where the mother has a severe hemophilia A gene. It aims to understand bleeding risks during pregnancy and childbirth for mothers, and how children develop antibodies to factor VIII treatment. By observing families over time, researchers …

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 at whether a quick bedside test can measure fibrinogen levels in trauma patients with severe bleeding within minutes, instead of the usual 30-45 minutes. Fibrinogen helps blood clot, and low levels can worsen bleeding. The test is being checked for feasibility an…

This study looks at how metamizole (Optalgin), a common painkiller, changes the levels of blood thinners (apixaban or rivaroxaban) in cancer patients. Researchers will measure drug concentrations at several time points in 30 adults. The goal is to understand if metamizole interfe…

This study looks at two types of blood thinners—direct oral anticoagulants (DOACs) and vitamin K antagonists (VKAs)—to see which works better at preventing repeat blood clots in people with a low-risk form of antiphospholipid syndrome (APS). APS is an autoimmune condition that ra…

This observational study aims to understand how endothelial colony-forming cells (ECFCs) behave in people with von Willebrand disease (VWD) and acquired von Willebrand syndrome (AVWS) compared to healthy individuals. Researchers will collect blood samples from 48 participants to …

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 looks at leftover blood samples from 100 patients having heart surgery to see how certain immune cells (monocytes) change during the procedure. The goal is to understand links between inflammation and blood clotting risks. No new treatments are being tested; it is pure…

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 reports on a single adult with severe hemophilia A who is receiving long-term anticoagulation (blood thinners). The goal is to see if newer factor VIII therapies can maintain safe clotting levels while preventing dangerous clots. Only one participant is enrolled, so th…

This study looks back at how antithrombin, a blood-clotting protein, was prescribed to 160 intensive care patients at Strasbourg University Hospitals over two years. Researchers want to see if these prescriptions were appropriate and how the drug was used in real-world settings. …

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 observes 80 transgender individuals starting cross-sex hormone therapy (testosterone or estrogen) to see how it changes eating behavior, metabolism, weight, and heart health. Participants are followed over time with no extra treatments or procedures. The goal is to und…

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 looks at how well a rapid blood test called TEG 6s measures clotting in healthy children. About 125 kids having planned surgery will give a small blood sample for the test. The goal is to find normal values for children and compare them to existing ranges. No treatment…

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 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 looks at people with atypical hemolytic uremic syndrome (aHUS), a rare disease that damages small blood vessels and kidneys. Researchers want to understand how certain rare gene mutations cause the disease by growing cells from blood samples in the lab. The goal is to …

This study looks at why cancer patients have a much higher risk of developing blood clots. Researchers will collect blood clots from 120 patients (with and without cancer) who undergo a procedure to remove clots. They will analyze the clots for cancer cells and other factors to b…

This observational study follows 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare and serious kidney condition. Researchers will collect safety and effectiveness data on patients treated with eculizumab or ravulizumab, as well as those receiving other care. The…

This registry collects health data from 4,000 children with hemophilia A or B across Europe. Researchers track bleeding episodes, joint health, and antibody development to clotting factors. The goal is to understand long-term outcomes and improve treatment for this rare bleeding …

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 follows 120 men with hemophilia A who have developed inhibitors (antibodies that block standard treatment). Researchers will observe how doctors manage their condition using different therapies, including immune tolerance induction and newer drugs like emicizumab. The …

This registry will collect detailed information from 50,000 patients who receive emergency blood transfusions during active bleeding while under anesthesia. The goal is to analyze how transfusions affect blood clotting, oxygen levels, and other vital signs. By studying this data,…

This study is testing new blood tests to see if they can better measure how well bleeding treatments work in people with haemophilia who have developed inhibitors (antibodies that block standard treatment). The researchers will take blood samples from 20 patients before and after…