Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

Study Overview
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Brief Summary
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This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.

Detailed Description
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This was a phase two pilot clinical trial designed to evaluate the safety and efficacy of the combination of deferasirox and deferoxamine in transfusion dependent thalassemia with a range of systemic iron burden. The duration of combined therapy was 12 months. Changes in liver iron concentration, ferritin, myocardial iron, and serum creatinine was monitored.

Official Title
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Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

Conditions
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Thalassemia, Iron Overload

Intervention / Treatment
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* Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Transfusion Dependent Thalassemia If iron between 5-15 mg/g dry liver by SQUID, subject must have a documented endocrinopathy or cardiac finding Older than 8 years Exclusion Criteria: Participating on another interventional clinical trial

Ages Eligible for Study
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Minimum Age: 8 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 (Exjade) and Deferoxamine (DFO)<br>All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline. | Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)<br>* All subjects will be given Deferasirox 20-30 mg/kg for 7 days per week. All subject will be given Deferoxamine 50 mg/kg for 3-7 days per week. The number of days for Deferoxamine will be determined by liver iron concentration at baseline.<br>* Other names: Deferoxamine (DFO);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months | Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline. | 12 months | 
| Change in Serum Creatinine During 12 Months Combined Chelation Therapy | Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Thalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overload