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

This treatment has been approved for sale to the public.
Sponsor:
Collaborator:
ApoPharma
Information provided by (Responsible Party):
Alan Cohen, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT00293098
First received: February 16, 2006
Last updated: February 8, 2012
Last verified: February 2012
  Purpose

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.


Condition Intervention
Iron Overload
Drug: deferiprone

Study Type: Expanded Access     What is Expanded Access?
Official Title: Compassionate Use of Deferiprone in Patients With Thalassemia and Iron-Induced Heart Disease

Resource links provided by NLM:


Further study details as provided by Children's Hospital of Philadelphia:

Study Start Date: March 2006
Intervention Details:
    Drug: deferiprone
    oral administration of 75 mg/kg/day in three divided doses, usually in combination with deferoxamine therapy
    Other Name: Ferriprox
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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00293098

Locations
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
ApoPharma
Investigators
Principal Investigator: Alan R Cohen, MD Children's Hospital of Philadelphia
  More Information

No publications provided

Responsible Party: Alan Cohen, Chair, Department of Pediatrics, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00293098     History of Changes
Other Study ID Numbers: 2006-2-4700
Study First Received: February 16, 2006
Last Updated: February 8, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital of Philadelphia:
Iron overload
Thalassemia
Iron induced heart disease
deferoxamine (Desferal)
Deferiprone

Additional relevant MeSH terms:
Heart Diseases
Thalassemia
Iron Overload
Cardiovascular Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 25, 2014