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.

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

Official Title
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1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

Conditions
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Myelodysplastic Syndrome, Thalassemia

Intervention / Treatment
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* Drug: Deferasirox


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age ≥ 2 years 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) ECOG Performance Status ≤ 2 Transfusion overload confirmed with ferritin level >1000 µg/l. No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure). Serum creatine level > ULN No proteinuria Liver enzymes level < 5 ULN. No pregnancy or lactation Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents. Exclusion Criteria: Age < 2 years No iron overload (Ferritin level <1000 µg/l). Primary iron overload (hereditary hemochromatosis) Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure) Elevated serum creatinine > ULN or/and proteinuria Liver enzymes level >5 ULN. Pregnancy or lactation. Other protocol-defined inclusion/exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 2 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 |
| --- | --- | --- |
| changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade |  | Baseline assessment is followed by monthly assessments for up to 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination |  | Baseline assessment is followed by monthly assessments for up to 1 year. | 
| changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade |  | at baseline and 1 at year (at the end of study). | 
| Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination. |  | From the start of study up to 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload