Beta thalassemia

Researchers are testing a stem cell transplant that uses low-dose radiation and two immune-suppressing drugs instead of high-dose chemotherapy. The goal is to cure severe sickle cell disease and beta-thalassemia in adults up to age 65 who have a matched sibling donor. Participant…

This trial tests a modified stem cell transplant for people with severe sickle cell disease or beta-thalassemia who are at higher risk of transplant failure. The approach uses low-dose radiation and a mix of immunosuppressive drugs to help the body accept donor stem cells without…

This study looks at growth problems in children with thalassemia, a blood disorder. Researchers will test a program that includes health education, nutrition plans, exercise, and support to help kids grow better. The goal is to improve height, weight, and overall well-being in 36…

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 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 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 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 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 trial investigates whether a 12- to 36-week program of aerobic and strength exercises can improve body composition, muscle function, and bone density in adolescents and adults with thalassemia. Participants will follow a self-directed routine of 30 minutes of exercise, 5 day…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…