Beta-thalassemia and related diseases

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 whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

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 study tests a new drug called SNH-119014 in 30 adults with a type of thalassemia that does not require regular blood transfusions. The goal is to see if the drug is safe and helps improve anemia. Participants will be monitored for side effects and how their body responds to …

This study compares transfusions using red blood cells from umbilical cord blood versus standard adult donor blood in extremely premature infants. About 176 babies born before 28 weeks or weighing under 1000 grams will be enrolled. The goal is to see if cord blood reduces the ris…

This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12-35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it is safe and can help patients produce enough healthy red blood cells to stop needing transf…

This early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

This early-phase trial will test a single dose of a new drug called YOLT-204 in 18 children and teens with transfusion-dependent β-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce the need for blood transf…

This Phase 3 trial tests whether two antioxidant supplements, N-acetyl cysteine and alpha-lipoic acid, can improve blood vessel health and reduce oxidative stress in adults with beta thalassemia. Sixty-six participants will take one of the supplements or a placebo alongside their…

This study tests whether motivational interviewing, a type of counseling, can help adults with beta-thalassemia major follow their iron chelation therapy more consistently. Forty-five participants in Oman will receive three one-on-one sessions with a nurse over six to eight weeks…

This study will test if a special oral health education program can improve the oral hygiene and quality of life of 45 children aged 8-10 with β-thalassemia major. The program teaches proper brushing and dental care. Researchers will measure changes in oral cleanliness and how ch…

This study will measure levels of a small molecule called microRNA-155 in 100 people who need regular blood transfusions, mostly due to beta thalassemia. The goal is to see if higher levels of this molecule are linked to the formation of harmful antibodies against donated blood. …

This study looks at children with beta-thalassemia major, a genetic blood disorder requiring lifelong transfusions. Researchers will measure growth and check for risk factors like iron overload that may stunt development. No new treatment is given—just careful observation of 85 c…

This study looks at how thalassemia patients in Greece feel day-to-day while taking a standard iron-removing medicine called deferasirox. Researchers will survey 150 adults to measure their physical and mental well-being. The goal is to understand real-world quality of life, not …

This study will measure certain hormones in 80 boys aged 10 to 18 who have transfusion-dependent beta thalassemia. The goal is to see how iron buildup from repeated blood transfusions affects the glands that control puberty and fertility. By checking levels of inhibin B, INSL3, a…

This study looks at body composition—fat, muscle, and bone mass—in children aged 5 to 18 with beta thalassemia major who receive regular blood transfusions. Researchers will use a simple test to measure these body components and see how they relate to transfusion frequency, iron …