Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001203
Status:
COMPLETED
Last Update Posted:
2019-12-12
First Post:
1999-11-03
Brief Title:
Deferoxamine for the Treatment of Hemochromatosis
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Clinical Course of Patients With Transfusional Hemochromatosis on Deferoxamine
Status:
COMPLETED
Status Verified Date:
2015-11-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
1 To determine if deferoxamine given regularly can indefinitely prevent the heart liver and endocrine complications associated with transfusional hemochromatosis
2 To determine whether heart disease caused by transfusional hemochromatosis can be reversed by intensive treatment with deferoxamine
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
1 To determine if deferoxamine given regularly can indefinitely prevent the heart liver and endocrine complications associated with transfusional hemochromatosis
2 To determine whether heart disease caused by transfusional hemochromatosis can be reversed by intensive treatment with deferoxamine
Detailed Description:
The purposes of this protocol are two-fold 1 to determine whether deferoxamine given subcutaneously on a regular basis can indefinitely prevent the cardiac endocrine and hepatic complications of transfusional hemochromatosis and 2 to determine whether cardiac disease can be reversed by intensive intravenous treatment in patients who already have objective evidence of cardiac dysfunction The clinical manifestations and course of patients who require regular blood transfusions is well established Those with congenital anemias who require transfusions from birth develop cardiac disease in their teens and their mean of survival is only 17 years Adults with acquired anemias begin to exhibit cardiac manifestations of iron deposition after 100-200 units of packed red cells Deferoxamine when given by the subcutaneous route has been shown to reduce substantially the total iron burden in thalassemic patients Our results indicate that cardiac complications are delayed or prevented We plan to continue to follow our cohort of patients on optimal medical management to determine if chelation alters disease outcome Patients with heavy iron burdens who already manifest cardiac disease will be chelated intensely to determine whether reducing the iron burden is associated with reversal of cardiac complications
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 85-H-0087 | None | None | None |