Clinical Trial: Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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
Brief 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.
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: To evaluate the effects of treatment on the liver iron content(LIC)
Original Primary Outcome:
Current Secondary Outcome:
- 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
- Evaluate the relationship between PD and safety variables
Original Secondary Outcome:
Information By: Novartis
Dates:
Date Received: June 3, 2003
Date Started: May 2003
Date Completion:
Last Updated: March 2, 2011
Last Verified: March 2011
