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The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox | Drug |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the effects of treatment on the liver iron content(LIC) |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate tolerability profile | ||
| Estimate the absolute and relative change of LIC and total body iron excretion (TBIE) rate | ||
| Evaluate the relationship between LIC and potential surrogate markers |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Oakland | Oakland | California | 94609 | United States | ||
| Stanford Hospital |
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| Evaluate the relationship between PD and safety variables |
| Stanford |
| California |
| 94305-5208 |
| United States |
| Northwest Medical Specialists | Arlington Heights | Illinois | 60004 | United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| Weill Medical College of Cornell University | New York | New York | 10021 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104-4318 | United States |
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| D009190 | Myelodysplastic Syndromes |
| D005198 | Fanconi Syndrome |
| D029503 | Anemia, Diamond-Blackfan |
| D000741 | Anemia, Aplastic |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001855 | Bone Marrow Diseases |
| D015499 | Renal Tubular Transport, Inborn Errors |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D029502 | Anemia, Hypoplastic, Congenital |
| D012010 | Red-Cell Aplasia, Pure |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D000080983 | Bone Marrow Failure Disorders |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
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