Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

Exclusion Criteria

• •Evidence of iron deficiency defined by Fe:TIBC ratio \<15%, or serum ferritin \<12 ng/mL;
• •Treatment with deferiprone in the past 12 months;
• •Previous failure of treatment with deferiprone, or previous discontinuation of treatment with deferiprone due to adverse events;
• •Conditions known to contraindicate the use of deferiprone (history of agranulocytosis or recurrent episodes of neutropenia);
• •A serious, unstable chronic illness not related to PKAN condition during the past 3 months before screening visit including but not limited to: hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease;
• •Evidence of abnormal liver or renal function (serum liver enzyme level(s) \> 3 times upper limit of normal at screening) or abnormal creatinine levels at screening visit;
• •Disorders associated with neutropenia (ANC \< 1.5 x 10\^9/L) or thrombocytopenia (platelet count \< 50 x 10\^9/L) in the 12 months preceding the initiation of the study medication. Exception: for patients whose neutropenia was attributed by the treating physician to episodes of infection or to drugs associated with a decline in the neutrophil count and in whom the ANC has fully recovered at the screening visit;
• •History of malignancy;