Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (a double-blind placebo-controlled trial in which participants were randomized to receive either deferiprone or placebo for 18 months) are eligible to enroll.
Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
TIRCON2012V1-EXT is a multi-center, single-arm, open-label study. All patients who completed the earlier study TIRCON2012V1 are eligible to take part. In the initial study, patients were randomized in a 2:1 ratio to receive 18 months of treatment with either the iron chelator deferiprone or placebo, respectively. In this extension study, all participants will receive deferiprone for 18 months. Thus, depending on which product was received earlier, patients will be on deferiprone for a total of either 1.5 years or 3 years. As in the earlier study, assessments will be carried out every six months to look at the safety of the drug and to see if patients are showing any improvement in dystonia and other symptoms of PKAN.
Pantothenate Kinase-Associated NeurodegenerationPKANNeurodegeneration with Brain Iron AccumulationNBIADeferiproneFerriproxPharmaceutical Solutions
You can join if…
Open to people ages 5 years and up
Completed study TIRCON2012V1
You CAN'T join if...
Withdrew from the study TIRCON2012V1 for reasons of safety
Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study
UCSF Benioff Children's Hospital Oakland Oakland, California, 94609, United States
Newcastle University Institute of Human Genetics Newcastle Upon Tyne, NE1 3BZ, United Kingdom
Klinikum der Universität München Munich, 80336, Germany
Foundation Neurological Institute C. Besta Milan, 20133, Italy