This study is investigating a new medication being developed as a potential treatment for Non-Transfusion Dependent Thalassemia (NTDT) or Thalassemia Intermedia. Thalassemia occurs when there is an abnormality or alteration in one of the genes involved in hemoglobin production. Hemoglobin is the protein molecule in red blood cells that carries oxygen. The disorder results in excessive destruction of red blood cells, which leads to anaemia. Anaemia is a condition in which the body does not have enough normal, healthy red blood cells.
Thalassemia is inherited, meaning that at least one parent must be a carrier of the condition.
There are two main forms of thalassemia: alpha-thalassemia and beta-thalassemia. In alpha-thalassemia, at least one of the alpha globin (protein) genes has an alteration or abnormality. In beta-thalassemia, the beta globin genes are affected. Beta-thalassemia comes in two forms, which are thalassemia major (requires regular blood transfusions) and thalassemia intermedia (don’t need blood transfusions). Current therapies to treat NTDT have limited effect and thus there is a need for new treatment options.