Beta thalassemia

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…