Acquired aplastic anemia

This phase II trial tests a vaccine designed to protect stem cell transplant recipients from cytomegalovirus (CMV), a common virus that can cause serious illness in people with weakened immune systems. The vaccine uses a modified, harmless virus to deliver three CMV proteins, tra…

This study tests a vaccine called Triplex to prevent cytomegalovirus (CMV) infection in adults who have received a stem cell transplant. CMV is usually harmless but can be life-threatening for people with weakened immune systems. The vaccine uses harmless virus pieces to train th…

This early-stage trial tests a new antibody drug called Hu8F4 in 72 people with advanced blood cancers like leukemia and myelodysplastic syndrome. The goal is to find the safest dose and see if it can slow or stop cancer growth. Researchers will also track how long patients live …

This study tracks 132 people with PNH who are taking Fabhalta to see how safe it is and what side effects occur. Researchers will monitor infections and check for serious red blood cell breakdown if the drug is stopped. The goal is to better understand the drug's risks in everyda…

This early-phase study tests whether adding a new drug called pelabresib to the standard treatment ruxolitinib is safe and helpful for Japanese adults with myelofibrosis, a type of bone marrow cancer. Only 6 people will take part, and the main goal is to check for side effects an…

This study follows about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking pegcetacoplan (Aspaveli/Empaveli) as part of their routine care. Researchers will track blood markers, symptoms, and side effects for up to 3 years to see how well the drug works out…

This phase II trial tests the drug decitabine in 21 patients with advanced myelofibrosis, a bone marrow cancer that causes scarring and anemia. The goal is to see if decitabine can improve blood counts and reduce the need for transfusions. Researchers are also monitoring side eff…

This early-stage trial tests two drugs—imetelstat and ruxolitinib—together in people with myelofibrosis, a type of bone marrow cancer. The study has two parts: first, finding a safe dose of the combination, and second, checking if it improves symptoms like fatigue and spleen disc…

This phase 3 trial tests crovalimab in about 50 people with PNH who have not used similar drugs before. PNH causes red blood cells to break apart, leading to fatigue, pain, and need for transfusions. Crovalimab is given as an IV infusion and then as injections under the skin to b…

This early-phase trial tests a reduced-intensity stem cell transplant using a half-matched donor and a drug called cyclophosphamide after the transplant to prevent rejection. The goal is to see if this approach is safe enough for patients with advanced myelofibrosis, a serious bo…

This study follows patients who have already been taking ruxolitinib alone or with panobinostat in earlier trials and are still benefiting. Researchers will track side effects over time to see how safe these drugs are for long-term use. About 296 participants with conditions like…

This study tests a pill called ruxolitinib for people whose immune system attacks their bone marrow, causing conditions like severe anemia. The drug aims to stop this attack and help the bone marrow recover. Participants take the pill twice daily for up to 6 months and are follow…

This study tests whether crovalimab works as well as eculizumab for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 210 adults who have not had prior complement inhibitor therapy will receive either drug. The main goals are to see if crovalimab…

This study tests whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant from a partially matched donor, can reduce serious infections in the first 100 days while still preventing graft-versus-host disease (a common complication where donor cells att…

This phase 3 trial is testing whether ropeginterferon alfa-2b, a biologic drug, can help control blood cell counts and symptoms in adults with early or low-risk primary myelofibrosis, a bone marrow disorder. Participants receive either the drug or a placebo every two weeks for up…

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 trial is testing an experimental drug called flonoltinib in people with intermediate to high-risk myelofibrosis, a type of bone marrow cancer that often causes an enlarged spleen. Participants will be randomly assigned to receive either a low or high dose of flonoltinib, or …

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 study looks at the long-term safety and tolerability of iptacopan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have already completed earlier phase 2 or 3 studies with the drug. About 208 participants will continue taking iptacopan and be monitored for side e…

This phase 3 trial compares two standard treatments for severe aplastic anemia (SAA) in patients up to age 25 who lack a matched sibling donor: unrelated donor bone marrow transplant (URD BMT) versus immune suppressive therapy (IST). The study measures which approach better preve…

This phase 3 trial compares crovalimab, a new injectable drug, to eculizumab in about 190 people with paroxysmal nocturnal hemoglobinuria (PNH) who are already on complement inhibitors. The study aims to see if crovalimab is as safe and effective, with a focus on side effects and…

This Phase 3 trial tests whether adding pelabresib to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a rare bone marrow disorder. About 430 adults who have not had prior JAK inhibitor treatment will take either the combination or …

This phase 1 trial tests whether the drug tagraxofusp can help prevent relapse in people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, myelofibrosis, or chronic myelomonocytic leukemia) who have recently received a stem cell transplant. Participant…

This study tests the long-term safety of an experimental drug called OMS906 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Participants receive OMS906 as an intravenous infusion every 8 weeks. The goal is to see if this dosing schedule is safe an…

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 study tests whether the drug luspatercept can help people with myelofibrosis-related anemia become independent of red blood cell transfusions. Participants are adults with myelofibrosis who are already taking a JAK inhibitor and need regular transfusions. The study c…

This early-stage study tests a new drug called IOA-244 in about 210 adults with advanced or metastatic cancers like lung cancer, melanoma, and lymphoma. The main goal is to see if the drug is safe and tolerable, both alone and combined with other treatments. Researchers also chec…

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 study looks at two treatment options for people with a high-risk form of myelofibrosis, a rare bone marrow cancer. Participants choose whether they want a stem cell transplant or the best non-transplant treatments available. Researchers then track survival and quality of lif…

This phase 2 study tests the drug selinexor against standard treatments in 112 people with myelofibrosis who have already tried JAK inhibitors. The main goal is to see if selinexor can shrink the spleen and improve symptoms. Participants are randomly assigned to receive either se…

This early-phase trial tests whether adding specially grown umbilical cord stem cells to a standard bone marrow transplant can improve outcomes for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. About 40 participants ag…

This study monitors about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking iptacopan (Fabhalta). Researchers will use registry data to count infections caused by certain bacteria. The goal is to better understand the real-world safety of this medic…

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 is testing the long-term safety and effectiveness of an experimental drug called XH-S003 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that can cause fatigue, blood clots, and other serious problems. About 26 people who have already ta…

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

This study provides extended access to the drug momelotinib for 237 people with a type of bone marrow cancer called myelofibrosis. Participants had already been taking momelotinib in earlier studies and had not seen their disease get worse. The goal is to see how safe the drug is…

This study follows about 80 people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken danicopan in a previous trial. They will continue taking danicopan as an add-on to their standard C5 inhibitor therapy. The goal is to see if the combination remains safe and …

This study follows 64 Polish adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking ravulizumab as part of their routine care. Researchers will track blood markers like LDH and how many patients need blood transfusions over time. The goal is to see if the drug works…

This early study tests whether elotuzumab can help people with a specific type of myelofibrosis, a serious bone marrow disorder. The drug targets a protein on cancer cells to try to improve blood counts, shrink an enlarged spleen, and ease symptoms. The trial includes 15 adults w…

This study was designed to test whether combining cyclosporine with drugs that boost platelet production (eltrombopag, hetrombopag, or romiplostim) could help people with newly diagnosed severe aplastic anemia who need blood transfusions. The trial was withdrawn before enrolling …

This study tests whether adding the drug itacitinib to the usual two-drug regimen (cyclophosphamide and tacrolimus) can better prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. GVHD is a serious complication where donor cells at…

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 phase 2 trial tests whether adding the drug ruxolitinib to standard care can prevent graft-versus-host disease (GVHD) in older adults with acquired aplastic anemia who receive a bone marrow transplant. GVHD is a common and serious complication where donor immune cells attack…

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 tests a new drug called BMS-986158, either alone or combined with standard treatments (ruxolitinib or fedratinib), in about 216 people with high-risk myelofibrosis, a type of blood cancer. The goal is to see if the drug is safe and can shrink an enlarged spleen, a comm…

This early-stage trial tests a new drug called VSA012 in about 50 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study includes people who have not used complement inhibitors before or who still have low hemoglobin …

This study tests a combination of two experimental drugs, pozelimab and cemdisiran, for people with PNH, a rare blood disease that destroys red blood cells. The goal is to see if this combo works better and is safer than current standard treatments (ravulizumab and eculizumab). A…

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 study tests a stem cell transplant method for people with blood cancers like leukemia or lymphoma. The approach uses chemotherapy, total body radiation, and a drug called cyclophosphamide after the transplant to prevent graft-versus-host disease, where donor cells attack the…

This study tests whether giving the drug ruxolitinib before, during, and after a donor stem cell transplant can prevent graft-versus-host disease (GVHD) in people with myelofibrosis. GVHD is a serious complication where donor cells attack the patient's body. The trial enrolls 53 …

This study tests whether combining ruxolitinib and thalidomide can shrink the cancer and improve symptoms in 30 people with myelofibrosis, a rare bone marrow disease. Participants include those who have not responded to prior treatment or are newly diagnosed with higher-risk dise…

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 study tests whether adding the drug selinexor to the standard treatment ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. About 353 adults who have not yet been treated for their condition will take either the combin…

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 study tests a new drug (WJ01024) combined with an existing drug (ruxolitinib) in 33 adults with intermediate- or high-risk myelofibrosis whose spleen is enlarged and who didn't respond well to or couldn't tolerate previous JAK inhibitor treatments. The goal is to see if the …

This study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…

This study follows 50 adults with PNH who take danicopan along with their usual medication (eculizumab or ravulizumab). Researchers track serious side effects and infections over time to see if the combination is safe for long-term use. Participants are already in a PNH registry …

This study looks at the long-term safety of danicopan when added to standard treatments (Soliris or Ultomiris) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will analyze data from 50 adult patients in an international registry, trac…

This early-phase study tests the safety of combining three drugs—navitoclax, venetoclax, and decitabine—for people with advanced myeloid blood cancers like leukemia and myelofibrosis. The goal is to find a safe dose that can control the disease. Sixteen adults aged 18 and older a…

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 the drug sirolimus can prevent severe aplastic anemia from coming back after patients stop taking cyclosporine. About 84 people aged 2 and older who have responded to initial treatment will be randomly assigned to either take sirolimus for 3 months or jus…

This study tests an experimental drug called LP-005 in 30 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The goal is to see if LP-005 can lower a key disease marker (LDH) and raise hemoglobin levels without needing bloo…

This study tests whether adding the experimental drug navitoclax to the standard drug ruxolitinib can shrink the spleen and reduce symptoms better than other available treatments in adults with myelofibrosis that has returned or not improved. About 330 participants will be random…

This early-phase trial tests a combination of two treatments for people whose myeloid blood cancer (like AML or MDS) has returned after a matched stem cell transplant. The treatments are a specially processed donor immune cell infusion and an immunotherapy drug called ipilimumab.…

This study looks at the long-term safety of a drug called HRS-5965 for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It includes 132 patients who previously took the drug and benefited from it. Researchers will track side effects and measure how we…

This study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…

This study tests a drug called fedratinib in Japanese adults with a serious bone marrow disease called myelofibrosis. The goal is to see if the drug can shrink an enlarged spleen and control the disease. About 31 people will take part, and the study will check safety and the best…

This program offers continued access to a combination of two drugs, pozelimab and cemdisiran, for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare condition where red blood cells break apart. It is for patients who have already completed a related study and aims to m…

This Phase 2 trial tests NTQ5082, an oral drug, in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells break apart. The drug blocks a part of the immune system to prevent this destruction. The main goal is to see if it can raise h…

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that can cause dangerous blood clots. Researchers want to know how often PNH appears in people under 50 who have had a stroke, mini-stroke, or brain vein clot with no clear cause. The study will screen 200 partici…

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 early-phase study tests whether vaccinating stem cell donors against cytomegalovirus (CMV) can prevent the virus from causing problems in blood cancer patients after a transplant. About 34 donor-recipient pairs are taking part. The vaccine is given to the donor before stem c…

This study evaluates whether a single dose of TQ05105, a JAK inhibitor being developed for myelofibrosis, affects the heart's electrical activity (QT interval) in healthy Chinese adults. Sixty participants receive TQ05105, placebo, and moxifloxacin (a positive control) in a cross…

This early-stage study in 16 healthy adults checks if taking a common stomach acid drug (rabeprazole) changes how the body absorbs a new experimental drug (TQ05105) being developed for myelofibrosis. Participants take TQ05105 alone and later with rabeprazole to compare drug level…

This study looks at how patients with advanced blood cancers (like acute myeloid leukemia) who are not eligible for a cure, along with their nurses and doctors, feel about the benefits of blood transfusions. Researchers will give questionnaires before each transfusion to compare …

This study follows about 40 people with intermediate or high-risk myelofibrosis who are already taking gecacitinib. Researchers will collect bone marrow and blood samples each year to see how the disease and gene mutations change over time. No new drugs are given—this is an obser…

This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease ou…

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…