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Summary

for people ages 5 years and up (full criteria)
at Oakland, California and other locations
study started
estimated completion:

Description

Summary

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.

Official Title

Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)

Details

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.

Keywords

Pantothenate Kinase-Associated Neurodegeneration PKAN Neurodegeneration with Brain Iron Accumulation NBIA Deferiprone Ferriprox Nerve Degeneration Pharmaceutical Solutions

Eligibility

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

Locations

  • 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

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
ApoPharma
ID
NCT02174848
Phase
Phase 3
Lead Scientist
Elliott Vichinsky
Study Type
Interventional
Last Updated
March 22, 2017