A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Study Overview
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Brief Summary
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Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.

Official Title
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A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Conditions
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Beta-Thalassemia, Hemosiderosis

Intervention / Treatment
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* Drug: deferasirox


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone Exclusion Criteria: Means levels of ALT > 300 U/L Serum creatinine above upper limit of normal Active hepatitis C or chronic hepatitis B receiving specific treatment

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
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deferasirox<br> | Drug: deferasirox<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver Iron Concentration (as measured by biopsy) |  | at baseline and after 1 yeor of ICL670 treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores) |  | after 1 year of ICL670 treatment | 
| Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation |  | monthly assessments for 1 year | 
| Adverse events |  | during 1 year ICL670 treatment | 
| hematology and biochemistry, urinalysis |  | monthly assessments for 1 year ICL670 treatment | 
| ECG and Echocardiography |  | 6-monthly for 1 year ICL670 treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone