Beta-thalassemia and related diseases

This trial tests whether giving defibrotide before and after a stem cell transplant can prevent a serious liver condition called sinusoidal obstructive syndrome (SOS) in children and young adults with sickle cell disease or beta thalassemia. Participants receive defibrotide start…

This study follows people who have already received an experimental gene therapy called BRL-101 for transfusion-dependent β-thalassemia, a severe blood disorder requiring regular transfusions. Researchers will monitor participants over the long term to check for safety issues, su…

This early-stage trial tests a gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. The approach uses the patient's own blood stem cells, which are edited to boost fetal hemoglobin—a healthy type that can replace the defective hemoglobin. Up to 1…

This study tests a stem cell transplant using cells from mismatched unrelated or partially matched family donors for people with severe sickle cell disease and other transfusion-dependent anemias. The approach removes certain immune cells (T-cells) from the donated stem cells to …

This study tests whether a combination of two drugs, thalidomide and hydroxyurea, can safely boost fetal hemoglobin levels in people with beta thalassemia major. The goal is to improve overall hemoglobin levels and reduce the need for regular blood transfusions. The trial include…

This study tests a drug called mitapivat in children aged 1 to 17 with alpha- or beta-thalassemia who need regular blood transfusions. The goal is to see if mitapivat can reduce the number of transfusions needed compared to a placebo. The study involves 54 participants and lasts …

This Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mi…

This clinical trial tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The transplant uses a lower-intensity conditioning regimen with alemtuzumab, low-dose radiation, and abatacept to help dono…

This study tests a single-dose gene therapy for people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular red blood cell transfusions. The therapy uses a patient's own stem cells, modified with a virus to produce healthy hemoglobin, and then infus…

This study tests a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a blood disorder that often requires regular transfusions. The goal is to see if the drug is safe and can reduce the need for transfusions or raise hemoglobin levels. The trial is still re…

This early-stage trial is testing the safety of fostamatinib, a drug already used for arthritis, in 25 adults with stable sickle cell disease. Participants take the pill twice daily for up to 6 weeks, with close monitoring for side effects. The goal is to see if the drug is safe …

This trial tests a second stem cell transplant using a gentler conditioning regimen of busulfan, fludarabine, and low-dose total body irradiation. It is for people with blood disorders, hemoglobin diseases, or immune deficiencies whose first transplant did not achieve enough dono…

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

This phase 2 trial tests a stem cell transplant from a donor for people with high-risk sickle cell disease, thalassemia, and other red blood cell disorders. Participants receive drugs and low-dose radiation before the transplant to help the new cells take hold. The study will tra…

This study is testing a stem cell transplant from half-matched (haploidentical) donors for people with sickle cell disease or beta-thalassemia. Before the transplant, patients receive drugs to lower their immune system, which may reduce the risk of serious side effects like graft…

This study is testing a drug called luspatercept in adults with a severe blood disorder called beta-thalassemia who need regular blood transfusions. The goal is to see if the drug can reduce how many transfusions they need and improve their hemoglobin levels. Researchers will fol…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for reg…

This study tests a special stem cell transplant for children and adults with blood cancers like leukemia. The transplant uses stem cells that have had certain immune cells removed to lower the risk of graft-versus-host disease, a serious complication. About 70 participants will r…

This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This Phase 2 study tests an oral drug called AND017 in 64 people with β-thalassemia, a blood disorder that often requires regular transfusions. Half the participants get the drug, half get a placebo, and everyone continues standard care. The goal is to see if AND017 is safe and c…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

This study offers a second stem cell transplant to people with sickle cell disease or beta-thalassemia whose first transplant is losing its effect. Participants receive donor stem cells along with drugs to help their body accept them. The goal is to restore donor cell levels and …

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 Phase 2 trial tests whether adding low-dose thalidomide to luspatercept can reduce the number of blood transfusions needed by adults with transfusion-dependent β-thalassemia. 78 participants will be randomly assigned to receive either the combination or luspatercept alone fo…

This early-stage trial is testing the safety of a drug called panobinostat in 18 adults with sickle cell disease. The drug aims to boost fetal hemoglobin and reduce inflammation, which may help control the disease. Participants take capsules three times a week for 12 weeks, and r…

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 trial tests an oral drug called SP-420 in about 90 adults with transfusion-dependent β-thalassemia or low-risk myelodysplastic syndromes (MDS). The goal is to see how well SP-420 removes excess iron from the liver and other organs, and whether it is safe. Participants take t…

This study follows 5 people with β-thalassemia who received CS-101, a gene-edited stem cell therapy, in an earlier trial. Researchers will monitor them for years to see if the treatment remains safe and allows them to stay free from blood transfusions. The goal is to understand w…

This early study tests a new type of stem cell transplant called 'super transplant' for children aged 7-12 with severe thalassemia. Unlike standard transplants, this one does not require chemotherapy beforehand and aims to avoid graft-versus-host disease. Only 5 participants will…

This study is working on a new blood test for pregnant women that can check for serious genetic conditions like cystic fibrosis, spinal muscular atrophy, and sickle cell disease. Researchers will collect blood samples from 4,000 pregnant women who are at higher risk of passing on…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

This study collects biological samples like blood, saliva, and urine from 300 adults aged 18-70, both with and without blood disorders such as sickle cell disease and thalassemia. The goal is to use these samples for research to better understand these conditions and related dise…

This study provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…

This study looks at how blood transfusions affect tiny molecules called peroxides in very premature babies. These molecules can damage cells if they build up. Researchers will measure peroxide levels before and after transfusions, and also use a special light device to check oxyg…

This study collects bone marrow that is normally thrown away during surgery from people with and without blood disorders like sickle cell disease. Researchers will use these samples in the lab to learn how to better manipulate stem cells for gene therapy and to study cell health.…

This study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …

This study aims to measure how long red blood cells survive in people with sickle cell disease, thalassemia, and other inherited blood disorders. Researchers will take a blood sample, label the red cells with biotin (a vitamin), and infuse them back into the participant. Over up …

This study looks at 350 adults with transfusion-dependent beta-thalassemia to find differences between those who have atrial fibrillation (a type of irregular heartbeat) and those who do not. Researchers will compare clinical tests, lab results, and imaging to better understand t…

This study is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…

This study looks at why people with transfusion-dependent beta thalassemia have trouble exercising. Researchers will compare 36 patients to healthy volunteers using bike tests, blood samples, and questionnaires. The goal is to find the physical and mental factors that limit their…