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

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Philadelphia
• Collaborator: ApoPharma
• Treatments: Deferiprone; Deferoxamine; Iron

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