Beta-thalassemia major

This trial compares an experimental herbal formulation called Irochel against the standard medication deferoxamine in managing iron overload caused by repeated blood transfusions in thalassemia patients. The study involves 132 children and adolescents aged 10 to 16 with transfusi…

This study tested whether giving children with transfusion-dependent thalassemia a combination of hydroxyurea and thalidomide works better than either drug alone. Ninety children aged 3-18 were split into three groups: one getting both drugs, one getting only hydroxyurea, and one…

This early-stage study tested a new gene-edited stem cell transplant in 2 children with severe β-thalassemia, a serious blood disorder requiring lifelong transfusions. The treatment aims to help the body produce healthy red blood cells and reduce or stop the need for transfusions…

This phase 2 trial tested whether combining thalidomide and hydroxyurea can reduce the need for blood transfusions in people with transfusion-dependent beta thalassemia. The study enrolled 100 patients over age 2 in Pakistan, where thalassemia is common and life expectancy is low…

This pilot study tested a stem cell transplant from closely matched unrelated donors for 8 patients with severe sickle cell disease or thalassemia major. The donated stem cells were specially processed to remove certain immune cells to reduce the risk of rejection and graft-versu…

This completed Phase 4 trial tested two drugs—hydroxyurea and metformin—in 96 people with transfusion-dependent beta-thalassemia. Participants took one of the two drugs for a year. Researchers measured changes in fetal hemoglobin levels and how often blood transfusions were neede…

This study tested a new treatment called ET-01 in 3 people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular blood transfusions. The treatment uses the patient's own stem cells, which are modified and then transplanted back. The goal was to see i…

This early-stage study tested a single dose of a gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The main goals were to check safety and see if the treatment helps the body make more healthy hemoglobin. …

This study tested a once-weekly injection called dulaglutide (a GLP-1 drug) against daily insulin in 80 children aged 10-18 with transfusion-dependent thalassemia and diabetes. The goal was to see if the weekly shot could control blood sugar just as well or better than insulin ov…

This study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…

This study looked at 100 children with beta-thalassemia major and iron overload to see if simple blood tests (ferritin and hepcidin) can detect liver scarring (fibrosis) as accurately as other non-invasive methods. Researchers measured these blood markers and compared them to ult…

This study looked at how long donor red blood cells survive in children with sickle cell disease who get regular blood transfusions. Researchers labeled a small part of the transfused blood with a harmless marker and tracked it over time. The goal was to better understand why som…

This study tested a new, simpler method to measure iron buildup inside red blood cells of people with beta thalassemia major. The goal was to find a cheaper and easier way to monitor iron overload compared to current tests. Researchers analyzed blood samples from 100 patients to …