A multi-center, placebo controlled, double-blind trial comparing the efficacy and safety of
18 months of treatment with deferiprone versus placebo in patients with PKAN.
This investigator-initiated trial was funded by the European Commission's Seventh Framework
Programme (FP7/2007-2013, HEALTH-F2-2011, grant agreement No. 277984) to the TIRCON
consortium (Treat Iron-Related Childhood-Onset Neurodegeneration) and by the FDA Office of
Orphan Products Development (OOPD) (Dr. Elliott Vichinsky).