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