Phase I/II, Randomized Blinded Safety Trial of Deferiprone in NBIA/PKAN

Investigator: Christopher P. Hess, MD, PhD
Sponsor: Oakland Children's Hospital Medical Center

Location(s): United States


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.(Pantothenate Kinase-Associated Neurodegeneration)