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Be Part of Research - Trial Details - Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
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Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

Completed

Open to: ALL

Age: 4.0 - N/A

Newcastle Upon Tyne

Medical Conditions

Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration

This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


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.

This investigator-initiated trial was funded by the European Commission's Seventh Framework Programme (FP7/2007-2013, HEALTH-F2-2011, grant agreement No. 277984) to the TIRCON consortium (Treat Iron-Related Childhood-Onset Neurodegeneration) and by the FDA Office of Orphan Products Development (OOPD) (Dr. Elliott Vichinsky).

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Dec 2012 Oct 2016

Publications

"Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buchner B, Steele HE, Horvath R, Chinnery PF, Basu A, Kupper C, Neuhofer C, Kalman B, Dusek P, Yapici Z, Wilson I, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E. Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study. Lancet Neurol. 2019 Jul;18(7):631-642. doi: 10.1016/S1474-4422(19)30142-5."; "31202468"

INTERVENTIONAL

Intervention Type : DRUG
Intervention Description : Deferiprone 80 mg/mL oral solution will be administered twice daily (b.i.d.) for 18 months. An initial dose of 5 mg/kg b.i.d. will be administered for 6 weeks. The dose will then be escalated to 10 mg/kg b.i.d. and finally to 15 mg/kg b.i.d.

Intervention Arm Group : Deferiprone;

Intervention Type : DRUG
Intervention Description : A deferiprone matching placebo oral solution will be given twice daily for 18 months.

Intervention Arm Group : Placebo;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Newcastle University Institute of Human Genetics
    Newcastle Upon Tyne
    NE1 3BZ


The study is sponsored by ApoPharma and is in collaboration with Food and Drug Administration (FDA).



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Read full details for Trial ID: NCT01741532
Last updated 27 June 2019

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