Ignite Creation Date:
2024-05-05 @ 4:41 PM
Last Modification Date:
2024-10-26 @ 9:23 AM
Study NCT ID:
NCT00293098
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2012-02-09
First Post:
2006-02-16
Brief Title:
Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease
Sponsor:
Childrens Hospital of Philadelphia
Organization:
Childrens Hospital of Philadelphia
Study Overview
Official Title:
Compassionate Use of Deferiprone in Patients With Thalassemia and Iron-Induced Heart Disease
Status:
APPROVED_FOR_MARKETING
Status Verified Date:
2012-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Patients who have iron overload due to chronic blood transfusions and have developed heart failure or who are at high risk of heart failure because of the high levels of iron in their hearts will be treated with deferiprone an investigational drug in combination with deferoxamine Desferal Some studies suggest that deferiprone may be better than deferoxamine in removing iron from the heart and improving heart function and that using both drugs together may remove more iron Participants would make a clinic visit for lab studies each week and would continue to take deferiprone for as long as their physician feels it is useful in their care
Detailed Description:
Repeated red cell transfusions lead to transfusional iron overload because the body lacks an efficient mechanism to excrete excess iron Without treatment iron accumulates in the liver heart and endocrine glands Cardiac complications including arrhythmias and congestive heart failure are the most common cause of death from transfusional iron overload New magnetic resonance imaging MRI T2 techniques enable an estimation of cardiac iron loading and allow patients at the highest risk of cardiac disease those with T2 10 ms to be identified For over 30 years deferoxamine has been the standard therapy However the mode of administration is cumbersome subcutaneous or intravenous infusion over 8 to 12 hours daily leading to poor compliance Thus cardiac disease and early mortality continue to be a significant problem in patients treated with chronic transfusions Treatment of cardiac complications involves intensifying therapy with deferoxamine including recommending intravenous administration over a period of 24 hours daily Deferiprone is an oral chelating agent not FDA approved for use in the United States Recent studies indicate that deferiprone is superior to deferoxamine in removing cardiac iron and reducing iron-induced cardiotoxicity The most serious side effect of deferiprone is agranulocytosis and other side effects are gastrointestinal symptoms reversible arthralgia reddish discoloration of urine and rare cases of autoimmune disease Patients on the study will be closely monitored for these toxicities Patients who are currently regularly followed at The Childrens Hospital of Philadelphia will be prescribed deferiprone at 75 mgkgday in three divided doses taken orally in combination with deferoxamine at the patients current dose Labs will be drawn once per week to monitor neutrophil count with additional labs every three months to monitor ferritin and ALT levels
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