Beta thalassemia

This trial tests whether a person's own blood stem cells, modified in the lab to carry a corrected gene, can cure beta thalassemia. Participants receive one or more infusions of these gene-modified cells after a mild conditioning regimen. The goal is to see if the cells safely pr…

This phase 2 trial tests a one-time gene therapy called HGI-001 in people with transfusion-dependent β-thalassemia, a severe blood disorder that requires regular blood transfusions. The therapy takes the patient's own blood stem cells, modifies them outside the body to produce he…

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 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 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 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…