Blood platelet disease

This study tests a new drug called bomedemstat against the best available treatments for people with essential thrombocythemia (a blood disorder causing too many platelets) who did not respond well to or couldn't tolerate the standard drug hydroxyurea. About 340 adults will be ra…

This trial compares the drug ruxolitinib against the best available standard treatments for people with high-risk polycythemia vera (PV) or essential thrombocythemia (ET), two rare blood cancers that cause overproduction of blood cells and can lead to clots, bleeding, and severe …

This phase 1 trial is testing an experimental pill called flonoltinib maleate in people with certain bone marrow tumors, including myelofibrosis, polycythemia vera, and essential thrombocythemia. The drug targets two proteins involved in abnormal blood cell growth. The study aims…

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

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

This phase 2 trial tests whether fecal microbiota transplantation (FMT) — transferring gut bacteria from a healthy donor — can help adults with chronic immune thrombocytopenia (ITP) whose disease has not responded to or has relapsed after steroid treatment. Participants will rece…

This study is testing an experimental drug called povetacicept in adults with autoimmune cytopenias — conditions where the immune system mistakenly destroys blood cells, leading to low platelet counts (immune thrombocytopenia) or anemia (autoimmune hemolytic anemia and cold agglu…

This phase 3 trial compares the biologic drug P1101 (ropeginterferon alfa-2b) against the standard drug anagrelide in people with essential thrombocythemia, a rare blood cancer that causes too many platelets. The study enrolls adults who have not responded well to the first-line …

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

This study is a long-term extension of an earlier trial, testing the safety of a drug called RAY121 in people with six different immune system disorders, including antiphospholipid syndrome and immune thrombocytopenia. Participants have already completed four doses of RAY121 and …

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

This early-phase trial tests a combination of three drugs—azacitidine, venetoclax, and pevonedistat—in adults newly diagnosed with a challenging form of acute myeloid leukemia (AML). The goal is to find the best dose and see if the combo can improve remission rates. About 40 part…

This phase 3 trial tests a drug called imetelstat against the best available treatments for people with advanced myelofibrosis whose disease has stopped responding to JAK inhibitors. The study includes 327 participants and aims to see if imetelstat can help them live longer and f…

This trial tests a combination of two drugs, sirolimus and all-trans retinoic acid (ATRA), for people with autoimmune anemia that has not responded to standard treatments. Autoimmune anemia occurs when the immune system attacks red blood cells, causing fatigue and other symptoms.…

This study tests an experimental drug called LY2784544 in people with three types of blood cancers known as myeloproliferative neoplasms: polycythemia vera, essential thrombocythemia, and myelofibrosis. The trial enrolls patients who have not responded to, cannot tolerate, or hav…

This phase II trial tested whether adding veliparib (a targeted drug) to standard chemotherapy (topotecan and carboplatin) helps people with advanced myeloproliferative disorders, acute myeloid leukemia, or chronic myelomonocytic leukemia. The study enrolled 25 adults whose cance…

This study tests whether adding ianalumab to standard eltrombopag treatment helps adults with immune thrombocytopenia (ITP) who did not get better with steroids. ITP causes low platelet counts, raising bleeding risk. The trial involves 152 participants and measures how long the t…

This phase 3 trial tests whether adding ianalumab (a biologic drug) to standard corticosteroids helps adults with newly diagnosed immune thrombocytopenia (ITP) achieve a stable platelet response off all treatments at 12 months. About 226 participants will receive either ianalumab…

This study tests whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants receive the drug and are monitored for side effects and changes in blo…

This study tests a stem cell transplant from a matched unrelated donor for people with advanced blood cancers or blood disorders. Patients get chemotherapy and radiation before the transplant to prepare their body, then take drugs to prevent the donor cells from attacking their o…

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This study planned to test whether infusions of mesenchymal stem cells could safely help people with refractory immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial was withdrawn before enrolling any participants, so n…

This phase 2 study tests two drugs, bimagrumab and tirzepatide, alone or together, to see if they help adults with obesity or overweight lose weight safely. About 252 participants without type 2 diabetes will take the drugs or a placebo for up to 70 weeks. The main goal is to mea…

This phase 2 trial tests canakinumab, an anti-inflammatory drug, in 14 to 26 people with myelofibrosis, a rare bone marrow cancer that causes scarring, enlarged spleen, and fatigue. Participants receive injections every three weeks for about six months. The study checks if the dr…

This study was designed to test a radioactive drug (177Lu-PSMA-617) in men with advanced prostate cancer that has spread to the bone marrow and caused low blood cell counts. The goal was to see if the treatment was safe and how it affected the cancer. However, the study was withd…

This study looks back at how doctors in Germany prescribed caplacizumab (Cablivi) for acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood clotting disorder. Researchers will analyze data from 350 patients to see how well the drug works outside of clinical trials and…

This phase 3 study tests a drug called pacritinib against standard treatments chosen by the doctor in people with myelofibrosis who have very low platelet counts (below 50,000). The goal is to see if pacritinib can shrink the spleen and reduce symptoms like tiredness, pain, and i…

This phase 2 trial tests an experimental drug called ianalumab in 41 adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. Participants have already tried steroids and another standard treatment. The study mea…

This study tests whether adding eltrombopag to standard immunosuppressive therapy can improve blood cell counts in people with untreated severe aplastic anemia. Participants receive horse-ATG and cyclosporine plus eltrombopag for up to 6 months. The goal is to see if this combina…

This phase 2 trial tests whether isatuximab can help people whose blood cell counts stay low after a stem cell transplant for blood cancer. The study enrolls 12 adults whose disease is in remission but who have developed immune-related blood cell deficiencies. Participants receiv…

This study tests a medicine called TAK-755 in people with a rare inherited blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP). The condition causes dangerous blood clots and bleeding. TAK-755 replaces a missing enzyme to help prevent or treat these episod…

This early-stage trial tests an experimental drug called INCB057643, given alone or with the standard drug ruxolitinib, in about 140 adults with myelofibrosis or other advanced blood cancers. The main goal is to check safety and side effects, while also measuring if the drug can …

This phase 3 trial tests an oral drug called rilzabrutinib in 232 adults and adolescents with persistent or chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. Participants receive either rilzabrutinib or a placebo…

This study tests an oral medication called azacitidine (ONUREG) for people with a low- or intermediate-risk form of myelodysplastic syndrome (MDS), a bone marrow disorder. About 230 participants will receive either the drug or a placebo, along with standard supportive care. The g…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study tests whether adding diammonium glycyrrhizinate (a liver-protecting compound) to standard high-dose dexamethasone can improve platelet counts in adults newly diagnosed with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing b…

This study tests a drug called efgartigimod given as a shot under the skin for adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. The goal is to see if the drug is safe and helps maintain safe platele…

This phase 2 trial tests an experimental daily pill called IMG-7289 in 4 adults with essential thrombocythemia or polycythemia vera who did not improve on standard therapy. The drug targets the LSD1 protein to help control high blood cell counts. Researchers are checking whether …

This early-phase trial tests an experimental antibody called HMB-001 in 57 adults with Glanzmann thrombasthenia, a rare bleeding disorder. The study aims to see if the drug is safe and can reduce the number and severity of bleeds when given regularly. Participants receive single …

This study was designed to give people who had a stem cell transplant and developed low blood cell counts (anemia, low platelets) continued access to the drug fostamatinib. Only those who responded well in a prior study were eligible. The goal was to see if they could maintain st…

This study is for adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding and bruising. It tests the long-term safety and benefit of an oral drug called rilzabrutinib. Only 4 participants who already benefited fr…

This study aimed to test two drugs, bimagrumab and tirzepatide, alone or together, for helping adults with obesity or overweight and type 2 diabetes lose weight. It was planned for about 13 months but was withdrawn before enrolling any participants. The main goal was to see how m…

This study was designed to test the safety and tolerability of a new drug called HMPL-523 (sovleplenib) in adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding problems. The trial was withdrawn before enrolling any pa…

This phase 2 trial tests an experimental drug called IMG-7289 in 9 adults with essential thrombocythemia, a rare blood disorder causing too many platelets. The drug aims to lower platelet counts and ease symptoms. Participants take a daily pill, with doses adjusted individually. …

This trial is testing a new imaging tracer called FLT, which is used with a PET scan to measure how fast tumors are growing. The study includes people with various cancers or those awaiting a biopsy for a suspicious mass. The goal is to see if FLT PET imaging can help find and di…

This study is testing a new, faster blood test for diagnosing acquired thrombotic thrombocytopenic purpura (aTTP), a rare but life-threatening blood clotting disorder. The new test, called HemosIL AcuStar, will be compared to two existing tests in 500 patients from Spain and Port…

This pilot study tested three different strengths of inhaled cannabis (2.5%, 5%, and 10% THC) against a placebo to see if they could quickly relieve migraine headaches. Twenty adults with migraine took part in a crossover design, meaning each person tried all options. The main go…

This study screens up to 10,000 people with blood disorders or healthy volunteers to see if they can safely join other active research studies at the National Heart, Lung, and Blood Institute. Participants undergo clinical, imaging, and lab tests. If no suitable study is availabl…

This study aims to measure damage to tiny blood vessels in people with thrombotic thrombocytopenic purpura (TTP), both during active disease and in remission. Researchers will use brain MRI, eye scans, and nailfold capillary exams to detect abnormalities. 79 participants will be …

This study aimed to collect medical information from people who developed unusual blood clots within four weeks of receiving a COVID-19 vaccine. The goal was to better understand any possible link between the vaccine and these rare events. However, the study was withdrawn before …

This study looks at whether adding simple family history questions to a standard bleeding questionnaire can better identify children who truly have mild bleeding disorders. Researchers will review data from 1,000 children seen at a clinic since 2019. The goal is to see if this co…

This study looks at whether young adults (ages 21-25) who use high-potency cannabis concentrates can improve their memory and thinking by switching to less potent products. Researchers will test participants' cognitive performance and collect blood and urine samples. The goal is …

This study examines whether people with certain blood cancers (myeloproliferative neoplasms) have autoantibodies that block a key immune protein called type I interferon. These autoantibodies may increase the risk of severe COVID-19. Researchers will test blood samples from 219 p…

This study is collecting information on quality of life and symptoms from 572 Italian patients with certain types of blood cancers called myeloproliferative neoplasms (MPNs). Participants fill out questionnaires about how their disease and treatments affect their daily functionin…