Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Last updated on April 2022Recruitment
- 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 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 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 need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening
- 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 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.
Exclusion Criteria
- HIV positivity
- Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
- Platelet count <75×109/L, absolute neutrophils count < 1.5×109/L at Screening.
- ...
- HIV positivity
- Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
- Platelet count <75×109/L, absolute neutrophils count < 1.5×109/L at Screening.
- Hepatocellular disease such as hepatitis B (presence of HBs antigen), hepatitis C (presence of HCV RNA), liver cirrhosis.
- ALT (SGPT) levels >5 times ULN at Screening.
- Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
- Estimated MDRD < 30 mL/min/1.73 m2 at Screening.
- Splenectomy prior to or planned during the study
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 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 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 need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening
- 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 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.
Exclusion Criteria
- HIV positivity
- Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
- Platelet count <75×109/L, absolute neutrophils count < 1.5×109/L at Screening.
- ...
- HIV positivity
- Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
- Platelet count <75×109/L, absolute neutrophils count < 1.5×109/L at Screening.
- Hepatocellular disease such as hepatitis B (presence of HBs antigen), hepatitis C (presence of HCV RNA), liver cirrhosis.
- ALT (SGPT) levels >5 times ULN at Screening.
- Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
- Estimated MDRD < 30 mL/min/1.73 m2 at Screening.
- Splenectomy prior to or planned during the study
Tracking Information
- NCT #
- NCT02049450
- Collaborators
- Not Provided
- Investigators
- Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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