Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
- Sponsor
- Novartis Pharmaceuticals
- Study ID
- NCT02049450
- Phase
- PHASE2
- Status
- Completed
Conditions
- Thalassemia Major
Eligibility Criteria
- Sex
- ALL
- Age
- 18 Years - N/A
- Healthy Volunteers
- Not accepted
Interventions
- ruxolitinib — DRUGRuxolitinib was taken at a starting dose of 10 mg twice daily with dose adjustments within the range of 5 to 25 mg twice daily.
Study Details
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.
Key Dates
- Start date
- May 28, 2014
- Status verified
- Jun 2017
- Primary completion
- Apr 12, 2016
- Completion
- Apr 12, 2016
Study Design
- Enrollment
- 30 participants (actual)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: INC424 (ruxolitinib) - Study TreatmentRegularly transfused adult patients with thalassemia and spleen enlargement.
Primary Outcome Measure
Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC) [ Time Frame: week 6 to week 30 interval ]