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

NCT00293098 | Unknown

• 1 other identifier: 2006-2-4700
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Iron Overload

Keywords

Iron overload; Thalassemia; Iron induced heart disease; deferoxamine (Desferal); Deferiprone

Interventions

deferiprone DRUG

oral administration of 75 mg/kg/day in three divided doses, usually in combination with deferoxamine therapy

Also known as: Ferriprox

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• ApoPharma: collaborator

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Iron Overload; Thalassemia

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Alan R Cohen, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chair, Department of Pediatrics

Study Record Dates

• First Submitted: February 16, 2006
• First Posted: February 17, 2006
• Study Start: March 1, 2006
• Last Updated: February 9, 2012

Record last verified: 2012-02

Locations

US (1)