Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

• Status: Completed
• Trial end date: 2011-07-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload

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

Details

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

Criteria

Inclusion criteria:

• In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion

• Serum ferritin> 1800 µg/L

• Age: 18-80 years

• men and women

• Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells

• Deferoxamin therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason

• Eastern Cooperative Oncology Group (ECOG) performance status score between 0-2

• written informed consent

Exclusion criteria:

• beta thalassaemia minor,

• haemosiderosis caused by other than chronic transfusional iron overload,

• patients with impaired renal function (Creatinin clearance< 60 ml/ min),

• pregnancy,

• lactation,

• patient of childbearing potential unwilling to use contraceptive precautions

• known hypersensitivity to deferasirox or any ingredients,

• impaired hepatic function (SGOT,SGPT 5x above UNL).

• Patients severely ill due to underlying disease progression or other severe concomitant disease.

• Patients with poor prognosis of karyotype

• patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition

• History of nephrotic syndrome

• Significant proteinuria

• Patients with a previous history of clinically relevant ocular toxicity related to iron chelation

• Patients with positive test to HIV

Other protocol-defined inclusion/exclusion criteria may apply