This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

• Status: Completed
• Trial end date: 2011-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.

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

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Deferasirox; Iron

Criteria

Inclusion Criteria:

1. Age ≥ 2 years

2. Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)

3. ECOG Performance Status ≤ 2

4. Transfusion overload confirmed with ferritin level >1000 µg/l.

5. No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).

6. Serum creatine level > ULN

7. No proteinuria

8. Liver enzymes level < 5 ULN.

9. No pregnancy or lactation

10. Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.

Exclusion Criteria:

1. Age < 2 years

2. No iron overload (Ferritin level <1000 µg/l).

3. Primary iron overload (hereditary hemochromatosis)

4. Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)

5. Elevated serum creatinine > ULN or/and proteinuria

6. Liver enzymes level >5 ULN.

7. Pregnancy or lactation.

Other protocol-defined inclusion/exclusion criteria may apply