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

Main Inclusion Criteria:

• Males or females 4 years of age and older at screening visit;
• Have PKAN, confirmed by genetic testing (supporting evidence required);
• BAD total score ≥ 3 at the screening visit;
• Patients who have Deep Brain Stimulation (DBS) systems or baclofen pumps in place will be eligible for the study, but they must have had a stable setting for at least two months prior to the screening visit and stimulation parameters / pump settings must remain stable for the duration of the trial:

Main 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 10E9/L) or thrombocytopenia (platelet count < 50 x 10E9/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;

Other protocol inclusion or exclusion criteria may apply.