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Sponsored by: |
Weill Medical College of Cornell University |
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Information provided by: | Weill Medical College of Cornell University |
ClinicalTrials.gov Identifier: | NCT00738413 |
Subjects with thalassemia major require regular transfusion therapy to sustain life. The iron present in the transfused blood remains in the body where it can cause a variety of organ dysfunctions. Lifelong iron chelation therapy is needed to maintain iron balance but its effectiveness varies greatly.
Like that of deferoxamine (Desferal, DFO) the mainstay of chelation therapy for 30 years, the effectiveness of deferasirox (Exjade, ICL670), the newly approved, orally effective iron chelating drug, is not satisfactory in all subjects. Even with good compliance, the iron excretion induced by a given drug exhibits wide subject-to-subject variability. There is often persistent iron overload of extra hepatic tissues such as the heart and pancreas leading to cardiac disease and diabetes. Combining the drugs may be a better approach in those subjects at increased risk. The iron balance studies proposed will permit an assessment of the potential of such a combination to place subjects in net negative iron balance and the relative effectiveness of the combination in relation to that of the individual drugs, an additive effect being expected. With such information, physicians will be able to design individualized chelation regimens that maximize effectiveness while minimizing side effects by adjusting the ratio and/or the dosing schedule of the two drugs.
Condition | Intervention | Phase |
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Thalassemia |
Drug: Deferoxamine Drug: Deferasirox |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Active Control, Crossover Assignment, Safety/Efficacy Study |
Official Title: | An Iron Balance Study Comparing Deferasirox, Deferoxamine and the Combination of Both Drugs |
Estimated Enrollment: | 6 |
Study Start Date: | August 2008 |
Estimated Study Completion Date: | December 2009 |
Estimated Primary Completion Date: | September 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Arm 1: Active Comparator
Subjects will be treated for 6 days with deferoxamine.
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Drug: Deferoxamine
Deferoxamine will be administered subcutaneously over 8 hours for 6 days at a dose of 40 mg/kg.
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Arm 2: Active Comparator
Subjects will be treated for 6 days with deferasirox.
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Drug: Deferasirox
Deferasirox will be orally administered at a dose of 30 mg/kg once daily for 6 days.
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Arm 3: Experimental
Subjects will be treated for 6 days with a combination of deferoxamine and deferasirox.
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Drug: Deferoxamine
Deferoxamine will be administered subcutaneously over 8 hours for 6 days at a dose of 40 mg/kg.
Drug: Deferasirox
Deferasirox will be orally administered at a dose of 30 mg/kg once daily for 6 days.
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Ages Eligible for Study: | 18 Years to 50 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Subjects must have no clinically significant finding in their medical history, on physical examination or as a result of laboratory assessments other than those consistent with thalassemia major and its complications, such as compensated cirrhosis, endocrine insufficiency and diabetes.
Exclusion Criteria:
Contact: Robert W Grady, Ph.D. | 212-746-3422 | rwgrady@med.cornell.edu |
Contact: Patricia J Giardina, M.D. | 212-746-3415 | pjgiard@med.cornell.edu |
United States, New York | |
Weill Cornell Medical Center | Recruiting |
New York, New York, United States, 10021 | |
Contact: Robert W Grady, Ph.D. 212-746-3422 rwgrady@med.cornell.edu | |
Contact: Kristen Muirhead, BS 212-746-3264 krm2066@med.cornell.edu | |
Principal Investigator: Robert W Grady, Ph.D. | |
Sub-Investigator: Patricia J Giardina, M.D. |
Principal Investigator: | Robert W Grady, Ph.D. | Weill Cornell Medical College |
Responsible Party: | Weill Cornell Medical College ( Robert W. Grady, Ph.D. ) |
Study ID Numbers: | 0804009771, DK55463 |
Study First Received: | August 18, 2008 |
Last Updated: | August 19, 2008 |
ClinicalTrials.gov Identifier: | NCT00738413 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Iron chelation Iron balance Secondary iron overload deferoxamine deferasirox |
Hematologic Diseases Deferasirox Anemia Anemia, Hemolytic Thalassemia Anemia, Hemolytic, Congenital Genetic Diseases, Inborn |
Hemoglobinopathies Neoplasm Metastasis Chelating Agents Iron Overload Hemoglobinopathy Iron Deferoxamine |
Molecular Mechanisms of Pharmacological Action Hematologic Diseases Deferasirox Anemia Iron Chelating Agents Anemia, Hemolytic Thalassemia |
Pharmacologic Actions Siderophores Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hemoglobinopathies Chelating Agents Deferoxamine |