- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00293098
Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease
February 8, 2012 updated by: Alan Cohen, Children's Hospital of Philadelphia
Compassionate Use of Deferiprone in Patients With Thalassemia and Iron-Induced Heart Disease
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.
Study Overview
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.
Study Type
Expanded Access
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- ADULT
- OLDER_ADULT
- CHILD
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Collaborators
Investigators
- Principal Investigator: Alan R Cohen, MD, Children's Hospital of Philadelphia
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
March 1, 2006
Study Registration Dates
First Submitted
February 16, 2006
First Submitted That Met QC Criteria
February 16, 2006
First Posted (ESTIMATE)
February 17, 2006
Study Record Updates
Last Update Posted (ESTIMATE)
February 9, 2012
Last Update Submitted That Met QC Criteria
February 8, 2012
Last Verified
February 1, 2012
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Metabolic Diseases
- Hematologic Diseases
- Genetic Diseases, Inborn
- Anemia
- Iron Metabolism Disorders
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Hemoglobinopathies
- Heart Diseases
- Iron Overload
- Thalassemia
- Molecular Mechanisms of Pharmacological Action
- Chelating Agents
- Sequestering Agents
- Iron Chelating Agents
- Deferiprone
Other Study ID Numbers
- 2006-2-4700
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Iron Overload
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Novartis PharmaceuticalsCompletedTransfusional Iron OverloadItaly
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ApoPharmaCompletedTransfusional Iron OverloadEgypt, Cyprus, Oman, Saudi Arabia, Turkey
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Novartis PharmaceuticalsCompletedChronic Iron OverloadGermany
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Novartis PharmaceuticalsNot yet recruiting
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Novartis PharmaceuticalsCompletedCardiac Iron OverloadTaiwan, Egypt, Thailand, Turkey, United Kingdom, Italy, Canada, Greece
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Assiut UniversityUnknownPlatelet Changes in Cases of Iron Overload
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ApoPharmaCompletedIron Overload Due to Repeated Red Blood Cell TransfusionsUnited States, Canada, Greece, Italy
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Assiut UniversityUnknownPlatelet Changes in Cases of Iron Overload
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ShireTerminatedIron Overload Due to Repeated Red Blood Cell TransfusionsCanada, United States, Thailand, Italy, United Kingdom
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ShireTerminatedPharmacokinetics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation TherapyIron Overload Due to Repeated Red Blood Cell TransfusionsUnited States, Canada, Lebanon, Italy, Egypt
Clinical Trials on deferiprone
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Consorzio per Valutazioni Biologiche e FarmacologicheEuropean CommissionCompletedChronic Iron OverloadItaly, Cyprus, Egypt
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ApoPharmaCompleted
-
ApoPharmaCompleted
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Imperial College LondonCompletedParkinson's DiseaseUnited Kingdom
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SocraTec R&D GmbHSocraMetrics GmbHCompleted
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ApoPharmaAlgorithme Pharma IncCompleted
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Chiesi Canada CorpCompletedIron Overload | Beta Thalassemia Major AnemiaEgypt, Indonesia
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ApoPharmaCompletedIron Overload Due to Repeated Red Blood Cell TransfusionsUnited States, Canada, Greece, Italy
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ApoPharmaCompletedFriedreich's AtaxiaBelgium, France, Italy, Spain
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Royal Brompton & Harefield NHS Foundation TrustCORDA, The Heart Charity; Apotex Inc.; The Cooley's Anemia Foundation,; The UK...UnknownBeta-ThalassemiaItaly