NCT02049450

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Thalassemia Major

Interventions

Type	Name	Description
DRUG	ruxolitinib	Ruxolitinib was taken at a starting dose of 10 mg twice daily with dose adjustments within the range of 5 to 25 mg twice daily.

Timeline

• Start date: 2014-05-28
• Primary completion: 2016-04-12
• Completion: 2016-04-12
• First posted: 2014-01-30
• Last updated: 2017-07-17
• Results posted: 2017-06-15

Locations

7 sites across 5 countries: Greece, Italy, Lebanon, Thailand, Turkey (Türkiye)