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 study tested whether a combination of ibrutinib (a targeted drug) and rituximab works better than standard chemotherapy (FCR) for younger patients with untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma. About 529 people took part. The main goal was t…

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 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 Phase 2 trial tests an experimental drug called REGN7999 in about 95 people with non-transfusion dependent beta-thalassemia who have too much iron in their bodies. The drug is given as a shot under the skin and aims to safely reduce iron levels in the liver. Researchers will…

This phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…

This study tests whether luspatercept works better than epoetin alfa for treating anemia in adults with a certain type of low-risk MDS who have not needed blood transfusions or prior anemia drugs. About 360 participants will receive one of the two drugs, and researchers will trac…

This study tests an experimental drug called AG-946 (tebapivat) in 87 adults with lower-risk myelodysplastic syndromes (MDS) who have anemia. The goal is to see if the drug can raise hemoglobin levels or help patients go without blood transfusions for at least 8 weeks. Participan…

This phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…

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 study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …

This phase 3 study tests whether mitapivat, an oral medication, can safely reduce the number of blood transfusions needed by adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo for 48 weeks. The main goal is to see if m…

This phase 3 trial tests whether the drug mitapivat can improve anemia and reduce fatigue in people with alpha- or beta-thalassemia who do not need regular blood transfusions. About 194 participants received either mitapivat or a placebo daily for 24 weeks. The main goal is to se…

This study tracks over 1,000 Japanese patients with renal anemia (low red blood cells due to kidney disease) who are taking molidustat tablets. Researchers are monitoring for serious side effects like blood clots, high blood pressure, and tumors, as well as checking if red blood …

This study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…

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 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 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 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 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 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-stage study tests a gene therapy for people with severe beta-thalassemia who need regular blood transfusions. The treatment modifies the patient's own blood stem cells to produce healthy hemoglobin, aiming to reduce or eliminate the need for transfusions. Six participa…

This study tests whether starting luspatercept (Reblozyl) at the maximum approved dose can help people with low-risk myelodysplastic syndromes (MDS) go without red blood cell transfusions for at least 8 weeks. About 106 participants who currently need regular transfusions will re…

This early-phase trial tests whether adding special immune cells called veto cells to a stem cell transplant can help donor cells grow in the patient without causing severe graft-versus-host disease (GVHD). The study includes 16 people with various blood cancers or bone marrow fa…

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 offers expanded access to a gene therapy for people with severe sickle cell disease. The treatment uses a modified virus to deliver genetic material that increases fetal hemoglobin, a healthy type that prevents sickling. Patients receive their own blood stem cells back…

This study planned to test the drug hetrombopag in people with non-severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The goal was to see if the drug could safely raise platelet counts. However, the trial was withdrawn before enrolling any …

This early-phase trial is testing a new gene therapy called CS-101 for people with beta-thalassemia, a blood disorder that often requires lifelong transfusions. The treatment uses the patient's own stem cells, which are modified in a lab to produce fetal hemoglobin, potentially r…

This phase 3 trial tests a drug called ianalumab against a placebo in 90 adults with warm autoimmune hemolytic anemia (wAIHA) who have already tried at least one treatment without success. The goal is to see if ianalumab can raise hemoglobin levels and keep them stable for at lea…

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 an experimental drug called M281 (Nipocalimab) in adults with warm autoimmune hemolytic anemia, a condition where the immune system attacks red blood cells. The trial compares M281 to a placebo to see if it can improve hemoglobin levels and reduce fatigue. About …

This study tests an experimental drug called rilzabrutinib in 22 adults with warm autoimmune hemolytic anemia (wAIHA), a condition where the immune system attacks red blood cells. Participants take the drug daily for up to 24 weeks, with an option to continue longer. The main goa…

This trial aimed to compare the safety and effectiveness of adding luspatercept to cyclosporine versus cyclosporine alone in adults newly diagnosed with non-severe aplastic anemia (NSAA). The goal was to see if the combination could improve blood cell counts better than the stand…

This study was designed to test whether romiplostim N01 could help people with aplastic anemia caused by chemotherapy or radiation therapy. The trial was withdrawn before enrolling any participants, so no results are available. It was planned as a small, early-stage study at a si…

This study was designed to test whether adding enarodustat to cyclosporine helps people with a type of aplastic anemia who need regular blood transfusions. The trial was withdrawn before enrolling any participants, so no results are available. It would have compared the combinati…

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 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 phase 2 trial compares a drug combo (ASTX727 plus iadademstat) against ASTX727 alone for people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The study aims to see if adding iadademstat improves complete response rates. However, the trial …

This phase 3 trial compares two treatments for people with a bone marrow disorder called myelodysplastic syndrome (MDS) who have low red blood cell counts (anemia). One group gets lenalidomide alone, the other gets lenalidomide plus epoetin alfa, a drug that stimulates red blood …

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 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 study tests whether a package of school meals, vegetable gardens, nutrition classes, iron supplements, and deworming can reduce anemia and underweight in 1500 adolescents aged 14-17 in Tanzania. Four schools get the program, two serve as controls. Researchers will also inter…

This study tests a drug called HMPL-523 (Sovleplenib) in people with warm antibody autoimmune hemolytic anemia, a condition where the immune system attacks red blood cells. About 111 adults will receive either the drug or a placebo to see if it improves hemoglobin levels and cont…

This study tests a new medicine, crovalimab, for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that destroys red blood cells. It involves healthy volunteers and people with PNH to check safety and how well the drug works. The goal is to control the disease, not …

This early-stage trial tests an experimental drug called INCB000928, given alone or with the standard drug ruxolitinib, for people with myelofibrosis who have severe anemia or need regular blood transfusions. The main goals are to check the drug's safety, find the right dose, and…

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 study tests a gene therapy that modifies a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that prevents sickling. The goal is to reduce or eliminate severe pain crises in people with sickle cell disease. The treatment involves chemotherapy to…

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 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 phase 2 trial tests the drug acalabrutinib in people with chronic lymphocytic leukemia (CLL) who also have a blood condition called autoimmune hemolytic anemia (AIHA) that has come back or not responded to treatment. The study aims to see if acalabrutinib can control the ane…

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 looks at the long-term safety and tolerability of the drug mitapivat in adults with stable sickle cell disease. Participants, who previously benefited from mitapivat in an earlier study, take the drug twice daily for up to 48 weeks, with an option to continue for anoth…

This study tests a drug called luspatercept in 6 adults with lower-risk myelodysplastic syndromes (MDS) or related blood cancers who have anemia. The goal is to see if the drug can reduce or eliminate the need for regular blood transfusions. Participants receive luspatercept plus…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

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 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 study tests whether giving pregnant women a daily vitamin B12 supplement (50 micrograms) from early pregnancy until six months after birth helps their children grow and think better. Researchers are enrolling 800 pregnant women in Nepal who are at risk of low B12 levels. The…

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 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 study tests a gene therapy for adults with β-thalassemia major, a severe blood disorder requiring frequent transfusions. Doctors take the patient's own stem cells, fix the faulty gene, and return the corrected cells via an IV. A low dose of busulfan prepares the …

This study tests a stem cell transplant using cells from a half-matched family donor, combined with low-dose radiation and drugs to prevent rejection, for adults with severe sickle cell disease or beta-thalassemia. The goal is to see if this gentler approach can replace the disea…

This study tests a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Ten participants will receive their own blood stem cells modified with a healthy gene to help produce normal red blood cells. The goal is to raise hemoglobin l…

This study follows 14 people with Fanconi Anemia who previously received RP-L102 gene therapy. Researchers will monitor their health for years to see if the treatment safely improves blood counts and reduces the need for a bone marrow transplant. The goal is to understand long-te…

This phase 2 trial tests whether the drug itacitinib, given with standard care, can prevent graft-versus-host disease (GVHD) in people who receive a donor stem cell transplant for blood cancers. GVHD happens when the donor cells attack the patient's body. The study includes 31 ad…

This study looks at the long-term safety of the drug pociredir in 50 adults with sickle cell disease who have already taken it and seen benefits in an earlier study. Participants will take pociredir once daily for up to 48 months. The main goal is to track side effects and other …

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 follows 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment…

This was a planned early-stage study of a new drug called TER-1754 for people with hereditary hemorrhagic telangiectasia (HHT), a condition that causes frequent nosebleeds and abnormal blood vessels. The trial aimed to test safety and find the right dose, but it was withdrawn bef…

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 two drugs, vorinostat and azacitidine, together in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal is to find the best dose and see if the combination is safe and effective. About 135 participants will take part in this early-…

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 study tests whether a person's own stem cells can be safely injected into the spinal fluid to treat multiple system atrophy (MSA), a rare and serious brain disease. About 30 adults aged 30-80 with MSA will receive the treatment. The main goal is to check for side effects, an…

This large Phase 3 trial is testing an oral drug called etavopivat in 450 people with sickle cell disease. The goal is to see if taking a pill once daily can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises compared to a placebo. Participants must ha…

This study tests whether adding a nutrition education program for caregivers to standard oral iron therapy improves hemoglobin levels in children aged 1-5 with iron deficiency anemia. 150 children will be split into two groups: one gets iron pills alone, the other gets iron pills…

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 looks at whether a hormonal IUS or birth control pills with iron are better at improving anemia in women. About 405 women with mild to moderate anemia who want contraception will be randomly assigned to one of the two methods and followed for 12-18 months. The main goa…

This study tested a drug called AG-348 in 20 adults with a type of thalassemia that doesn't require regular blood transfusions. The goal was to see if the drug could safely raise hemoglobin levels, which carry oxygen in the blood. Participants took the pill twice a day for up to …

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 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 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 bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well 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 is testing a liposomal iron supplement with vitamin C and fiber (LISEFEX®) in 74 Mexican adults with mild to moderate iron deficiency anemia. Researchers are observing how well the supplement raises hemoglobin levels and how acceptable it is to patients. The supplement…

This study tests whether a low dose of pazopanib, a drug currently used for cancer, can reduce severe nosebleeds and improve anemia in people with hereditary hemorrhagic telangiectasia (HHT). About 70 participants will take either the drug or a placebo daily for 24 weeks. Researc…

This study tests whether adding an antibody called briquilimab (JSP191) to a standard stem cell transplant can help more donor cells take root in people with sickle cell disease or beta-thalassemia. About 40 patients aged 13 and older will receive the antibody along with low-dose…

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 study tests an oral drug called vadadustat (Vafseo) for treating anemia in people with end-stage kidney disease who are on hemodialysis. About 2,200 adults will be randomly assigned to take vadadustat three times a week or continue standard injectable treatments. The goal is…

This phase 2 trial tests the drug zanubrutinib (Brukinsa) in 25 people with primary cold agglutinin disease, a rare condition where the immune system attacks red blood cells in cold temperatures, causing anemia and poor circulation. The study aims to see if the drug can reduce an…

This study tests whether adding Moringa leaf powder to standard iron pills can better improve hemoglobin levels in pregnant women with iron deficiency anemia. About 59 women in their third trimester will take either Moringa capsules plus iron or iron alone for 60 days. Researcher…

This study tests whether adding the drug abatacept to standard care can prevent graft-versus-host disease (GVHD) in children receiving stem cell transplants from unrelated donors. GVHD occurs when donor cells attack the patient's body, causing serious illness. The trial will enro…

This study tests a new pill, vadadustat, taken three times a week, against the usual injected medicine for anemia in people with kidney failure who are on dialysis. About 350 adults will take part to see if the pill can safely keep their red blood cell levels in a healthy range. …

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 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 Phase 3 trial tested a stem cell transplant using blood stem cells from a half-matched (haploidentical) donor in 21 people with various blood cancers. The goal was to see if this approach could help patients who don't have a fully matched donor. Participants received chemoth…

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 study follows 5 people with beta-thalassemia who already received an experimental gene-edited cell therapy called CS-101. Researchers will monitor them for years to see if the treatment remains safe and whether they can stay free from blood transfusions for at least 12 month…

This study follows 8 people with β-thalassemia who already received a one-time treatment called CS-101, which modifies their own blood cells to produce fetal hemoglobin. No new drug is given. Researchers will monitor them for years to see if they stay free of blood transfusions a…

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 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 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 program provides continued treatment with nipocalimab for people with warm autoimmune hemolytic anemia (wAIHA) who have already shown improvement in a previous study. Participants receive the drug by IV infusion every 2 or 4 weeks. The goal is to keep their hemoglobin levels…

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 early-stage trial tests a one-time gene therapy called BRL-101 for people with transfusion-dependent beta-thalassemia, a severe blood disorder. The treatment uses the patient's own stem cells, edited with CRISPR to boost healthy hemoglobin production. The goal is to see if a…

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 study tests a gene therapy approach for people with severe sickle cell disease. The treatment uses a modified virus to add a gene that boosts fetal hemoglobin, which can reduce sickling and symptoms. Ten participants will receive their own gene-corrected blood st…

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 phase 2 study tests the drug olaparib in 114 people with metastatic breast cancer whose tumors have mutations in certain DNA repair genes (like BRCA1, BRCA2, ATM, PALB2, and others). Olaparib is a PARP inhibitor that may kill cancer cells by blocking their ability to fix dam…

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 phase 2 trial tests a gene therapy called RP-L102 for children with Fanconi anemia subtype A, a rare genetic disorder that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and infuse the corrected cells back. The goal…

This study looks at whether storing red blood cells in a low-oxygen environment (called hypoxic storage) can improve transfusion care for adults with transfusion-dependent beta-thalassemia. Participants already receiving these specially stored blood cells as part of their routine…

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 study was designed to test the safety of an iron pill called ferric citrate in children aged 6 to 17 with iron deficiency anemia and chronic kidney disease (not on dialysis). The plan was to compare the iron pill to standard care over 24 weeks. However, the study was withdra…

This study is testing the safety of a drug called ATGAM in one patient with moderate to severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The patient receives ATGAM by IV for 4 days and is monitored for 24 weeks for side effects. The goal …

This study follows 8 people who received a one-time gene therapy (BIVV003 for sickle cell disease or ST-400 for beta-thalassemia) to see if it remains safe and effective over many years. Researchers will track serious side effects, survival, and blood markers like hemoglobin leve…

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 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 expanded access program tested HBOC-201 (Hemopure), a liquid oxygen carrier made from cow hemoglobin, in adults with life-threatening anemia who cannot receive blood transfusions. The goal was to improve oxygen delivery to tissues. Participants had very low hemoglobin levels…

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 study tests a drug called luspatercept in people with anemia caused by myelodysplastic syndromes (MDS) or beta-thalassemia who need regular blood transfusions. The goal is to see if the drug is safe and can reduce the number of transfusions needed. About 85 participants in I…

This study planned to test a daily pill called vadadustat for anemia in children with chronic kidney disease. It aimed to see if the pill could safely replace standard injections. However, the trial was withdrawn before enrolling any participants, so no results are available.

This study was designed to test a once-daily pill called vadadustat for treating anemia in children with chronic kidney disease. The goal was to see if the drug could safely raise red blood cell levels without the need for injections. However, the study was withdrawn before enrol…

This study tests a drug called luspatercept in 70 adults with a low-risk form of MDS that causes anemia. The goal is to see if the drug can reduce the need for red blood cell transfusions. Participants receive the drug and are monitored for safety and effectiveness over time.

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…

This study tests a new approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Patients receive a combination of umbilical cord blood and stem cells from a family member, with the cord blood chosen based on specific genetic markers rather than …

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests whether a smartphone app can accurately detect anemia by analyzing images of the lower eyelid. The app uses artificial intelligence to estimate hemoglobin levels without a blood draw. Researchers will compare the app's results to standard lab tests in 3,000 adult…

This study tests a small, swallowable capsule called PillSense that can detect blood in the upper digestive tract within about 10 minutes. Researchers want to see if a negative result from the capsule can safely predict that a hospitalized patient with anemia or a positive stool …

This study tests whether special lights and cameras can help find early signs of mouth cancer in people at high risk. About 338 participants with precancerous spots or conditions like Fanconi anemia will have their mouths examined with fluorescence imaging, which makes abnormal c…

This study tests whether a newer form of iron (liposomal iron) works better than standard iron drops to prevent iron deficiency anemia in premature babies. About 60 preterm infants born at or before 34 weeks will receive either daily standard iron, daily liposomal iron, or liposo…

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 looks at whether taking vitamin D and fish oil supplements can reduce the risk of developing anemia in people aged 60 and older. Researchers are following 2000 participants from a larger heart-health trial who did not have anemia at the start. They will compare anemia …

This pilot study tests whether a daily mindful music listening program is practical and acceptable for patients hospitalized after an allogeneic stem cell transplant. About 35 adults will listen to music for one hour each day using a web-based platform. The study measures how fea…

This study looks at whether adding art therapy to physical therapy can help children aged 5 to 18 recover emotionally and physically after a stem cell transplant. For two weeks, some children receive both art and physical therapy daily, while others get only physical therapy. Res…

This study looks at whether giving blood transfusions to patients who are anemic after leaving the intensive care unit (ICU) can help them feel less tired and recover better. About 346 adults from UK hospitals will be randomly assigned to either the usual care (transfusions only …

This study looks at whether acceptance and commitment therapy (ACT) can improve sleep for adults with sickle cell disease. Participants will have weekly video chats with a coach for 8 weeks and wear a wrist device to track sleep. The goal is to see if this approach is practical a…

This study tests whether adding methadone to usual morphine-based pain medicine reduces severe mouth pain in children aged 6–18 who are getting a stem cell transplant for leukemia, aplastic anemia, or similar conditions. The mouth pain is a common side effect of the chemotherapy …

This study looks at how well a new iron supplement made from fish (SalmoFer®) is absorbed by the body and whether it causes stomach discomfort. About 61 women aged 21 to 42 with low iron or mild anemia will take the supplement and have their iron levels checked through blood test…

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 creates a bank of blood, cheek cells, semen, urine, or nail clippings from up to 1,200 healthy adult volunteers. The samples are used by scientists for lab research only, such as studying immune function or developing tests. Donors are paid for their time and must be N…

This study tests whether offering online educational videos and mailed saliva genetic testing kits helps more first-degree relatives of people with BRCA mutations get tested, compared to standard care with a family letter. About 820 participants will be enrolled across several me…

This study is a global registry that will follow about 500 people with pyruvate kinase (PK) deficiency, a rare inherited anemia, for up to 9 years. Researchers will collect medical data to better understand the disease's natural history, treatments, and complications. No new drug…

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 gathers information from 1,200 pregnant people carrying twins that share a placenta (monochorionic twins). Researchers track complications like preterm birth and infant death to better understand these high-risk pregnancies. No new treatments are tested; the goal is to…

This study looks at how anemia changes blood flow in the smallest blood vessels (microcirculation) in patients having open surgery for gastric cancer. Researchers will compare 27 anemic patients with 27 non-anemic patients, all receiving standard goal-directed fluid therapy. They…

This study tests whether a tablet-based decision support system helps frontline health workers in India and Nepal provide better antenatal care. About 1,320 pregnant women will take part. The tool guides workers to check blood pressure, blood sugar, urine protein, and hemoglobin,…

This study follows 50 young men who had cancer or blood disorders as children and received treatments that can harm fertility. Some stored testicular tissue before treatment; others did not. Researchers will check their fertility through exams, ultrasounds, blood tests, and semen…

This study aimed to check if special fetoscopes are safe and work well for laser surgery in twin pregnancies with complications. The study was withdrawn before any participants were enrolled, so no results are available. It would have looked for device problems and side effects.

This study is a registry that collects information from about 429 adults with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS), a rare condition where the immune system attacks red blood cells in cold temperatures. Researchers will track symptoms, treatments, compl…

This study follows 60 people with lower-risk myelodysplastic neoplasms (MDS) who need regular red blood cell transfusions. Over 6 months, participants use a smartphone app to answer weekly quality-of-life questions and take photos of their fingernails or eyelids. Researchers aim …

This pilot study looks at why military personnel often have low iron levels during training. Six physically active adults will receive either erythropoietin or a placebo and then do a 90-minute loaded march. The goal is to find the best way to measure iron absorption after exerci…

This study examines whether SGLT2 inhibitors, a type of diabetes drug, can improve red blood cell production and heart/kidney function in people with chronic heart failure. Researchers will compare blood tests, heart ultrasound measures, and clinical outcomes between 85 patients …

This study looks at over 3,400 newborns who are not vigorous at birth (between 35 and 42 weeks). Doctors want to see if milking the umbilical cord (pushing blood into the baby) before clamping it leads to fewer cases of brain injury or death compared to clamping the cord right aw…

This study was designed to test a holistic education program for stress management in people from colonized English-speaking countries. It aimed to help with conditions like stress, PTSD, anxiety, and fatigue using pastoral counseling and self-care activities. However, the study …

This study looks at whether children with anemia take longer to recover from diabetic ketoacidosis (DKA), a serious diabetes complication. Researchers will review medical records of 150 children aged 1 to 18 who had DKA at one hospital. They will compare recovery times and hospit…

This study follows 100 women with breast or gynecologic cancers to see if their diet before chemotherapy is linked to levels of a protein called hepcidin, and whether hepcidin levels affect how much chemo they can receive. The goal is to understand if eating certain foods might h…

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 was designed to observe how patients with hereditary tyrosinemia type 1 (HT-1) in China respond to nitisinone treatment in everyday medical practice. It planned to track serious health events like liver problems or death. However, the study was withdrawn before enrolli…

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 looks at 200 newborns with anemia to compare three types of blood transfusions: irradiated, washed, and leukocyte-reduced red blood cells. The goal is to see which works best and how each affects brain oxygen levels and inflammation. Researchers hope the results will h…

This study aimed to see if iron deficiency in new mothers is linked to depression and poorer interactions with their babies. Researchers planned to compare iron-deficient and iron-sufficient mothers using video-recorded play sessions. However, the study was withdrawn before any p…

This study looks at how a program that helps families lift themselves out of poverty affects the growth and learning of children aged 0-9 in Uganda. Researchers will compare kids in families that received the program to those that did not, measuring things like height, anemia, th…

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 follows 1,304 healthy Indian adolescents aged 8–10 over time to see how their growth, bone health, and muscle development are linked to their parents' nutrition. Researchers aim to understand why malnutrition persists across generations and identify windows of opportun…

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…

This study was designed to see if a new monitoring algorithm could detect early signs of ovarian damage in women and girls who had cancer treatment. Researchers planned to compare a group using the new algorithm with a group that received standard care. However, the study was wit…

This study follows 36 patients with aplastic anemia who previously received romiplostim in earlier trials. Researchers will monitor blood cell counts and safety for up to 5 years to see if benefits last and to check for any new health issues. The goal is to better understand the …