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Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00000595
Recruitment Status : Completed
First Posted : October 28, 1999
Last Update Posted : November 26, 2013
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary:
To determine whether deferoxamine prevented the complications of transfusional iron overload.

Condition or disease Intervention/treatment Phase
Anemia (Iron-Loading) Beta-Thalassemia Hematologic Diseases Hemoglobinopathies Thalassemia Iron Overload Hemochromatosis Drug: deferoxamine Phase 2

Detailed Description:


The prognosis of congenital or long-term anemia was formerly limited by the complications of blood transfusion, splenectomy, or infection, problems now largely overcome by sophisticated clinical care. Lifespan is now determined by the rate of myocardial iron deposition, with death occurring from cardiac failure or arrhythmia, usually between the ages of 15 and 25. Endocrine complications and hepatic enlargement are also evident by this age. Deferoxamine increases urinary iron excretion and is the only chelator currently available for chronic administration. Daily administration of deferoxamine results in negative iron balance in most patients by the age of 10; this study was designed to determine whether the onset of cardiac complications was delayed and life prolonged by iron removal.

This trial began in 1978. Its forerunner was a study involving both deferoxamine and ascorbic acid. Although ascorbic acid promotes iron removal, its administration was followed by cardiac deterioration in several patients. In this study, patients receiving subcutaneous deferoxamine were randomized to receive either ascorbic acid or placebo, thereby providing a controlled test of this agent in treatment of iron overload. Sixty-five patients with homozygous beta-thalassemia participated in the long-term chelation trial. Of these, 49 were randomized to the ascorbic acid trial.

Several noninvasive techniques have been developed to evaluate organ function in iron-overloaded patients, thereby facilitating the assessment of chelation therapy. These techniques included chest x-rays, electrocardiograms, echocardiograms, and 24-hour Holter monitoring to assess cardiac function. Liver function was evaluated by standard liver function tests, CAT scan, and live biopsy. During the last six years of the study, hepatic iron stores were measured magnetically with a dual channel superconducting quantum-interference susceptomer. Endocrine function was also assessed by standard tests.


All patients received subcutaneous deferoxamine and iron removal was determined by measurement of serum ferritin and periodic non-invasive measurements of liver iron concentration. Clinical status was evaluated by non-invasive testing of cardiac and endocrine function.

The study completion date listed in this record was inferred from the last publication listed in the Citations section of this study record.

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Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Study Start Date : January 1978
Actual Study Completion Date : September 1994

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Males and females, 5 years or older, with transfusional hemochromatosis.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00000595

Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
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OverallOfficial: Neal Young Laboratory of Hematology, NHLBI
Layout table for additonal information Identifier: NCT00000595    
Other Study ID Numbers: 401
First Posted: October 28, 1999    Key Record Dates
Last Update Posted: November 26, 2013
Last Verified: May 2000
Additional relevant MeSH terms:
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Hematologic Diseases
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action