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Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.

Thalassemia Major Clinical Trial

Official title:
A Single Arm, Multicenter, Phase IIa Study to Explore the Efficacy and Safety of Ruxolitinib (INC424) in Regularly Transfused Patients With Thalassemia

Clinical Trial Summary

Patients with severe thalassemia (thalassemia major) present with severe anemia that requires life-long transfusion therapy, spleen enlargement that may lead 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 suggest that JAK2 inhibition, by reducing spleen size, may improve hemoglobin levels, thereby eliminating the need for splenectomy and reducing transfusion requirement and related iron overload.

Clinical Trial Description

n/a

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02049450
Study type Interventional
Source Novartis
Contact
Status Completed
Phase Phase 2
Start date May 2014
Completion date April 2016
View Details
See also
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