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Work Package 4

Title of Work Package: A randomized, double-blind, placebo-controlled trial of deferiprone in patients with PKAN

Objective: The objective of WP4 is to establish the safety and efficacy of iron-chelating deferiprone in PKAN patients by conducting an adequately powered, randomized, double-blind, placebo-controlled trial. Study duration will be 18 months which will allow for assessment of symptomatic as well as disease-slowing neuroprotective effects of deferiprone as compared to the placebo group. (For more information, please visit ClinicalTrials.gov.)


  • Completion and approval of the final study protocol: Based on our clinical study protocol synopsis and subject to feedback from the ethics committee, the final study protocol as well as patient information and consent forms will be prepared by the coordinator, in close collaboration with clinical TIRCON partners. After approval by participating partners, the final protocol and the patient forms will be submitted to the institutional review boards of participating partners and also discussed with EMA and FDA. Before recruitment of the first participant, the study will be registered in a primary registry that participates in WHO’s International Clinical Trial Registry Platform .
  • Recruitment of PKAN patients: Patients eligible for the study (as known from local cohorts, the consortium’s survey and patient advocacies) will be invited to a screening visit at one of the hospitals in our international clinical network.
  • Safety Monitoring: A Safety Monitoring Board will meet at regular intervals during the study to review safety data in a blinded manner. They will be provided on an ongoing basis with a description of any serious adverse events.


Data Safety Monitoring Board (DSMB) Memos regarding the safety of patients participation in the clinical trial: