Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
NCT00061763 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 175
Last updated 2017-08-22
Summary
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.
Conditions
- Beta-thalassemia
- Myelodysplastic Syndromes
- Fanconi Syndrome
- Anemia, Diamond-Blackfan
- Anemia, Aplastic
Interventions
- DRUG
-
Deferasirox
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Novartis Pharmaceuticals · Novartis Pharmaceuticals
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 2 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2003-05-31
- Primary Completion
- 2004-11-30
Countries
- United States
Study Locations
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