Efficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload

Study Overview
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Brief Summary
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The purpose of this study is to investigate the effects of iron chelation using deferasirox in patients who show signs of iron overload after an allogeneic stem cell transplantation The iron overload must be due to blood transfusions.

Official Title
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A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/d) in Patients Three to Six Months After Allogeneic Hematopoietic Cell Transplantation in Whom Iron Overload is Present

Conditions
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Iron Overload

Intervention / Treatment
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* Drug: Deferasirox


Participation Criteria
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Eligibility Criteria
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Inclusion criteria: Transfusional iron overload three to six months after HCT with no evidence of active inflammation History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs). Patients of either gender and age ≥ 18 years. Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraception , or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months. Exclusion criteria: Non-transfusion related iron overload Active malignancy Known active viral hepatitis or known HIV positiveness Mean levels of alanine aminotransferase (ALT) > 5x ULN Treatment with any iron chelator after transplantation Uncontrolled systemic hypertension Serum creatinine > 1.5 ULN and/or serum creatinine clearance < 60 ml/min History of nephrotic syndrome. Previous history of clinically relevant ocular or auditory toxicity related to iron chelation. Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment Pregnant or breast feeding patients. Other protocol-defined inclusion/exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 18 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 |
| --- | --- | --- |
| To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox |  | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the relationship between serum ferritin, transferrin (TRF) and transferrin saturation during the whole study. |  | 52 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hematopoietic stem cell transplantation, HCT, iron overload, chelators, ICL670, deferasirox, serum ferritin, LIC, transfusional hemosiderosis, blood transfusions, adults, iron chelation, 3-6 months, allogeneic hematopoietic stem cell transplantation