Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients

• Status: Terminated
• Trial end date: 2016-10-17
• Target enrollment: 0
• Participant gender: All

Summary

Evaluated Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Cyclosporine; Cyclosporins; Deferasirox; Immunosuppressive Agents

Criteria

Inclusion Criteria:

• Main diagnosis: aplastic anemia

• Absence of severe and/or uncontrolled comorbidities

• Confirmed iron overload (serum ferritin ≥ 1000 mkg/L)

• Serum creatinine is not higher than the upper limit of normal for the given age

• Absence of severe proteinuria. Protein/Creatinine ratio should be < 0.5 mg/mg

• Liver enzymes are < 5 ULN

• Completion of a scheduled cycle of immunosuppressive treatment program, with no severe infectious or generalized hemorrhagic complications

• WHO (ECOG) performance status ≤ 2

Exclusion Criteria:

• No signed informed consent form

• Patient is under 18 years old

• Severe concomitant condition

• Severe infectious and generalized haemorrhagic complication following regular planned cycle of programmed immune suppressive treatment.

• History of increased sensitivity to active substance and any other ingredient of the medicinal product.

• Creatinine clearance (CC) < 60 ml/min and/or creatinine concentration in blood serum is 2 or more times higher than upper limit of age normal by results of 2 tests at Visits 1 and 2.

• Severe liver disorders (class C by Child-Pugh scale).

• Patients with aplastic anaemia in which chelator treatment will be ineffective due to rapid progression of the disease.

• Significant proteinuria basing on protein creatinine ratio > 1.0 mg/ml in urine sample from second urination at Visits 1 and 2 (or as an alternative in 2 of 3 urine samples at screening);

• Rare hereditary disorders related to galactose intolerance, severe deficit of lactase or glucose-galactose malabsorption;

• Pregnancy, lactation;

• Level of liver enzymes higher than 5 upper limits of age normal at Visits 1 and 2.