Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:01 AM
Study NCT ID:
NCT00000595
Status:
COMPLETED
Last Update Posted:
2013-11-26
First Post:
1999-10-27
Brief Title:
Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Heart Lung and Blood Institute NHLBI
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2000-05
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:
To determine whether deferoxamine prevented the complications of transfusional iron overload
Detailed Description:
BACKGROUND
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
DESIGN NARRATIVE
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
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
DESIGN NARRATIVE
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
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: