Deferoxamine for the Treatment of Hemochromatosis
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|ClinicalTrials.gov Identifier: NCT00001203|
Recruitment Status : Completed
First Posted : November 4, 1999
Last Update Posted : December 12, 2019
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When patients receive repeated blood transfusions the level of iron in the patient s blood can rise. When iron is processed in the body a protein known as hemosiderin can begin collecting in the organs. If too much hemosiderin collects in the organs they can begin to malfunction. This condition is called transfusional hemochromatosis.
An organ of particular importance in transfusional hemochromatosis is the heart. Patients born with diseases requiring blood transfusions at birth begin to develop heart problems in their teens. These patients typically only live for 17 years. Adults that require transfusions can begin experiencing heart problems after 100-200 units of backed red blood cells.
Deferoxamine (Desferal) is a drug that binds to iron and allows it to be excreted from the body. It is the only effective way to remove iron from patients who have been overloaded with iron because of multiple transfusions. Previous studies have lead researchers to believe that deferoxamine, when given as an injection under the skin (subcutaneous), can be delay or prevent heart complications.
Researchers plan to continue studying patients receiving deferoxamine as treatment for the prevention of heart complications associated with repeated blood transfusions. In this study researchers will attempt;
- To determine if deferoxamine, given regularly, can indefinitely prevent the heart, liver, and endocrine complications associated with transfusional hemochromatosis
- To determine whether heart disease caused by transfusional hemochromatosis can be reversed by intensive treatment with deferoxamine.
|Condition or disease|
|Diabetes Mellitus Heart Disease Hemochromatosis Thalassemia|
|Study Type :||Observational|
|Actual Enrollment :||151 participants|
|Official Title:||Clinical Course of Patients With Transfusional Hemochromatosis on Deferoxamine|
|Study Start Date :||April 22, 1985|
|Study Completion Date :||November 9, 2015|
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|Ages Eligible for Study:||4 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- INCLUSION CRITERIA
Patients studied under this protocol will be at risk for or have evidence of significant excess tissue iron.
Most patients will be on regular blood transfusion secondary to either congenital or acquired anemia.
The majority of patients have homozygous beta thalassemia.
Patients with sickle cell anemia will be included only when there is an absolute indication for regular blood transfusions (e.g., a history of stroke).
Twenty to thirty adults with acquired anemia and good long-term prognosis will be accepted for study if chelation can be initiated early in their transfusion history (less than 30-50 units).
Such patients will be excluded from study if they have diabetes or cardiac disease due to another cause (coronary artery or valvular heart disease).
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 ClinicalTrials.gov identifier (NCT number): NCT00001203
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Griffin P Rodgers, M.D.||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|Responsible Party:||National Heart, Lung, and Blood Institute (NHLBI)|
|Other Study ID Numbers:||
|First Posted:||November 4, 1999 Key Record Dates|
|Last Update Posted:||December 12, 2019|
|Last Verified:||November 9, 2015|
Liver Iron Concentration
Glucose Metabolism Disorders
Endocrine System Diseases
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Iron Metabolism Disorders