Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Status:
Completed
Trial end date:
2016-04-12
Target enrollment:
Participant gender:
Summary
Patients with severe thalassemia (thalassemia major) present with severe anemia that required
life-long transfusion therapy, spleen enlargement that led to increased transfusion
requirement, and other serious complications as early death, growth retardation, bone
deformations and iron overload due to blood transfusions. Splenectomy can significantly
reduce transfusion requirement in thalassemia patients, but it is associated with an
increased risk of serious complications such as sepsis and thrombosis. Preliminary
preclinical and clinical data suggested that JAK2 inhibition, by reducing spleen size, could
improve hemoglobin levels, thereby eliminating the need for splenectomy and reducing
transfusion requirement and related iron overload.