Recruitment

Recruitment Status
Completed

Inclusion Criteria

Patients need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening
Patients with thalassemia on a regular and stable transfusion regimen (at least 2 RBC units within every 4-week interval for 24 weeks prior to Screening) and anticipated to receive the same transfusion regimen during the study.
Patients with spleen enlargement at Screening, defined as spleen palpable below the costal margin and spleen volume of ≥ 450 cm3 as confirmed by MRI (or CT scan in applicable patients).
Patients need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening
Patients with thalassemia on a regular and stable transfusion regimen (at least 2 RBC units within every 4-week interval for 24 weeks prior to Screening) and anticipated to receive the same transfusion regimen during the study.
Patients with spleen enlargement at Screening, defined as spleen palpable below the costal margin and spleen volume of ≥ 450 cm3 as confirmed by MRI (or CT scan in applicable patients).

Exclusion Criteria

Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
ALT (SGPT) levels >5 times ULN at Screening.
...
Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
ALT (SGPT) levels >5 times ULN at Screening.
Estimated MDRD < 30 mL/min/1.73 m2 at Screening.
Splenectomy prior to or planned during the study
Hepatocellular disease such as hepatitis B (presence of HBs antigen), hepatitis C (presence of HCV RNA), liver cirrhosis.
HIV positivity
Platelet count <75×109/L, absolute neutrophils count < 1.5×109/L at Screening.

Summary

Conditions
  • ?-thalassemia Major
  • Thalassemia Major
Type
Interventional
Phase
Phase 2
Design
  • Allocation: N/A
  • Intervention Model: Single Group Assignment
  • Masking: None (Open Label)
  • Primary Purpose: Treatment

Participation Requirements

Age
Between 18 years and 125 years
Gender
Both males and females

Inclusion Criteria

Patients need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening
Patients with thalassemia on a regular and stable transfusion regimen (at least 2 RBC units within every 4-week interval for 24 weeks prior to Screening) and anticipated to receive the same transfusion regimen during the study.
Patients with spleen enlargement at Screening, defined as spleen palpable below the costal margin and spleen volume of ≥ 450 cm3 as confirmed by MRI (or CT scan in applicable patients).
Patients need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening
Patients with thalassemia on a regular and stable transfusion regimen (at least 2 RBC units within every 4-week interval for 24 weeks prior to Screening) and anticipated to receive the same transfusion regimen during the study.
Patients with spleen enlargement at Screening, defined as spleen palpable below the costal margin and spleen volume of ≥ 450 cm3 as confirmed by MRI (or CT scan in applicable patients).

Exclusion Criteria

Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
ALT (SGPT) levels >5 times ULN at Screening.
...
Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
ALT (SGPT) levels >5 times ULN at Screening.
Estimated MDRD < 30 mL/min/1.73 m2 at Screening.
Splenectomy prior to or planned during the study
Hepatocellular disease such as hepatitis B (presence of HBs antigen), hepatitis C (presence of HCV RNA), liver cirrhosis.
HIV positivity
Platelet count <75×109/L, absolute neutrophils count < 1.5×109/L at Screening.

Locations

Bangkok, 10700
Palermo, PA, 90146
Athens, GR, GR-115 27
Beirut, 1107 2020
Izmir, 35040
...
Bangkok, 10700
Palermo, PA, 90146
Athens, GR, GR-115 27
Beirut, 1107 2020
Izmir, 35040
Istanbul, 34093
Milano, MI, 20122

Tracking Information

NCT #
NCT02049450
Collaborators
Not Provided
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals