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Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease

Primary Purpose

Iron Overload

Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
deferiprone
Sponsored by
Children's Hospital of Philadelphia
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Iron Overload focused on measuring Iron overload, Thalassemia, Iron induced heart disease, deferoxamine (Desferal), Deferiprone

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Transfusional iron overload Overt cardiac failure or significant arrhythmia, OR high risk of developing cardiac failure as determined by T2* < 10 ms by magnetic resonance imaging (MRI) Signed consent form Patient regularly followed at The Children's Hospital of Philadelphia Unwillingness to participate in, or lack of suitability for, a clinical trial providing similar therapy Exclusion Criteria: Previously treated with deferiprone and had severe adverse reactions necessitating discontinuation Receiving other investigational drugs Receiving other drugs known to cause neutropenia Unexplained occurrences of neutropenia in past two years Pregnant or breastfeeding; or want to become pregnant. Sexually active but unwilling to use reliable birth control Other conditions which, in the opinion of the investigator, would make patient unsuitable for enrollment

Sites / Locations

  • The Children's Hospital of Philadelphia

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
February 16, 2006
Last Updated
February 8, 2012
Sponsor
Children's Hospital of Philadelphia
Collaborators
ApoPharma
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1. Study Identification

Unique Protocol Identification Number
NCT00293098
Brief Title
Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease
Official Title
Compassionate Use of Deferiprone in Patients With Thalassemia and Iron-Induced Heart Disease
Study Type
Expanded Access

2. Study Status

Record Verification Date
February 2012
Overall Recruitment Status
Approved for marketing
Study Start Date
March 2006 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital of Philadelphia
Collaborators
ApoPharma

4. Oversight

5. Study Description

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 Children's Hospital of Philadelphia will be prescribed deferiprone at 75 mg/kg/day in three divided doses, taken orally, in combination with deferoxamine, at the patient's 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload
Keywords
Iron overload, Thalassemia, Iron induced heart disease, deferoxamine (Desferal), Deferiprone

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
deferiprone
Other Intervention Name(s)
Ferriprox
Intervention Description
oral administration of 75 mg/kg/day in three divided doses, usually in combination with deferoxamine therapy

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Transfusional iron overload Overt cardiac failure or significant arrhythmia, OR high risk of developing cardiac failure as determined by T2* < 10 ms by magnetic resonance imaging (MRI) Signed consent form Patient regularly followed at The Children's Hospital of Philadelphia Unwillingness to participate in, or lack of suitability for, a clinical trial providing similar therapy Exclusion Criteria: Previously treated with deferiprone and had severe adverse reactions necessitating discontinuation Receiving other investigational drugs Receiving other drugs known to cause neutropenia Unexplained occurrences of neutropenia in past two years Pregnant or breastfeeding; or want to become pregnant. Sexually active but unwilling to use reliable birth control Other conditions which, in the opinion of the investigator, would make patient unsuitable for enrollment
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alan R Cohen, MD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease

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