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

The European Union is funding a deferiprone clinical trial in the U.S. and four sites in Europe through a Seventh Framework Programme grant called Treat Iron-Related Childhood-Onset Neurodegeneration or TIRCON.

Additionally, the U.S. Food and Drug Administration is funding a portion of the study in the U.S. The pharmaceutical company, ApoPharma Inc., is providing the deferiprone and placebo for all of the sites and is the contract research organization (CRO) that will monitor the study.

Those taking part will have a two-in -three chance of getting deferiprone and a one- in- three chance of getting the placebo. Neither the participant, the pharmacist or the study team will know who is getting the drug or placebo during the 18-month study.

The study participants will be ages 4 to adult and must have PKAN disease, confirmed by genetic testing.

U.S. Study Site:

Children’s Hospital and Research Center Oakland
Nancy Sweeters, RN, PNP

(510) 428-3885 ext. 4151

See brochure for more detailed information on this trial.