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

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

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

Conditions
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Myelodysplastic Syndromes, Beta-Thalassemia

Intervention / Treatment
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* Drug: deferasirox


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deferasirox<br> | Drug: deferasirox<br> <br> * Other names: ICL670;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload |  | monthly during the therapy and at the end of the treatment (aftr 9 months therapy) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events |  | during the treatment (9 months) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox