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| Sponsor: | Novartis Pharmaceuticals |
|---|---|
| Information provided by: | Novartis |
| ClinicalTrials.gov Identifier: | NCT00061763 |
Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Beta-thalassemia Myelodysplastic Syndromes Fanconi Syndrome Anemia, Diamond-Blackfan Anemia, Aplastic |
Drug: Deferasirox |
Phase II |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine |
| Enrollment: | 175 |
| Study Start Date: | May 2003 |
| Primary Completion Date: | November 2004 (Final data collection date for primary outcome measure) |
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United States, California | |
| Children's Hospital Oakland | |
| Oakland, California, United States, 94609 | |
| Stanford Hospital | |
| Stanford, California, United States, 94305-5208 | |
| United States, Illinois | |
| Northwest Medical Specialists | |
| Arlington Heights, Illinois, United States, 60004 | |
| United States, Massachusetts | |
| Children's Hospital Boston | |
| Boston, Massachusetts, United States, 02115 | |
| United States, New York | |
| Weill Medical College of Cornell University | |
| New York, New York, United States, 10021 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104-4318 | |
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
| Responsible Party: | External Affairs, Novartis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT00061763 History of Changes |
| Other Study ID Numbers: | CICL670A0108 |
| Study First Received: | June 3, 2003 |
| Last Updated: | March 2, 2011 |
| Health Authority: | United States: Food and Drug Administration |
|
beta-thalassemia iron overload deferoxamine Myelodysplastic Syndromes |
Fanconi Syndrome Anemia, Diamond-Blackfan Anemia, Aplastic |
|
Anemia Anemia, Aplastic Beta-Thalassemia Fanconi Syndrome Fanconi Anemia Myelodysplastic Syndromes Preleukemia Thalassemia Iron Overload Anemia, Diamond-Blackfan Hematologic Diseases Bone Marrow Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies |
Genetic Diseases, Inborn Kidney Diseases Urologic Diseases Renal Tubular Transport, Inborn Errors Metabolism, Inborn Errors Metabolic Diseases Anemia, Hypoplastic, Congenital DNA Repair-Deficiency Disorders Precancerous Conditions Neoplasms Iron Metabolism Disorders Red-Cell Aplasia, Pure Deferoxamine Deferasirox Siderophores |