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Sponsors and Collaborators: |
National Heart, Lung, and Blood Institute (NHLBI) Thalasemmia Clinical Research Network (TCRN) |
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Information provided by: | National Heart, Lung, and Blood Institute (NHLBI) |
ClinicalTrials.gov Identifier: | NCT00115349 |
The purpose of this study is to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and decreased ejection fractions. Secondary aims include evaluating changes in myocardial iron burden using T2* and estimating the relative incidence and severity of chelator-induced toxicity.
Condition | Intervention | Phase |
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Cardiovascular Diseases Heart Diseases Beta-Thalassemia |
Drug: Deferoxamine Drug: Deferiprone (L1) |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Thalassemia Clinical Research Network - Cardiac L1/DFO Trial |
Enrollment: | 20 |
Study Start Date: | June 2005 |
Study Completion Date: | October 2008 |
Primary Completion Date: | July 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
Deferoxamine (DFO) and deferiprone (L1) combination therapy
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Drug: Deferoxamine
Deferoxamine will be given daily for 12-24h/day 7 days a week either subcutaneous or intravenous at up to 50-60 mg/kg/day.
Drug: Deferiprone (L1)
The dose of L1, 75mg/kg in three divided oral doses, is the maximum dose at which toxicity has been tested in prospective trials
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2: Active Comparator
Deferoxamine (DFO) monotherapy
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Drug: Deferoxamine
Deferoxamine will be given daily for 12-24h/day 7 days a week either subcutaneous or intravenous at up to 50-60 mg/kg/day.
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DESIGN NARRATIVE:
Participants will be randomized to 1 year of treatment with L1/DFO combination therapy or DFO monotherapy. At baseline, 6 months, and 1 year on therapy, cardiac function will be assessed by MRI measurement of left ventricular ejection fraction (LVEF), T2*, Holter monitoring, and electrocardiography. Additional monitoring for safety includes weekly blood testing, monthly visits, and periodic eye and ear exams.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, California | |
Children's Hospital | |
Oakland, California, United States, 94609 | |
Children's Hospital of Los Angeles | |
Los Angeles, California, United States, 90027 | |
United States, Illinois | |
Children's Memorial Hospital | |
Chicago, Illinois, United States, 60614-3394 | |
United States, Massachusetts | |
Children's Hospital | |
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, 19104-4399 |
Principal Investigator: | John Porter, MD | University College, London |
Study Chair: | Patricia J. Giardina, MD | Weill Medical College of Cornell University |
Study Chair: | Ellis J. Neufeld, MD | Boston Children's Hospital |
Study Chair: | Elliott P, Vichinsky, MD | Children's Hospital and Research Institute, Oakland |
Study Chair: | Sonja McKinlay, Ph.D. | New England Research Institutes, Inc. |
Responsible Party: | National Institutes of Health ( National Heart, Lung, and Blood Institute ) |
Study ID Numbers: | 181, U01 HL65232, U01 HL65238, U01 HL65239, U01 HL65244, U01 HL65260 |
Study First Received: | June 21, 2005 |
Last Updated: | November 18, 2008 |
ClinicalTrials.gov Identifier: | NCT00115349 |
Health Authority: | United States: Food and Drug Administration |
Heart Diseases Hematologic Diseases Deferiprone Beta-thalassemia Anemia Anemia, Hemolytic Thalassemia Anemia, Hemolytic, Congenital |
Thalassemia minor Genetic Diseases, Inborn Beta-Thalassemia Hemoglobinopathies Hemoglobinopathy Iron Deferoxamine |
Molecular Mechanisms of Pharmacological Action Iron Chelating Agents Cardiovascular Diseases |
Chelating Agents Pharmacologic Actions Siderophores |