Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
Completed · Phase 3 · Has a placebo group
Conditions studied: Pantothenate Kinase-Associated Neurodegeneration
In brief
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).
Key facts
- Study ID
- NCT01741532
- Run by
- ApoPharma
- People needed
- 89
- Starts
- 2012-12-13
- Expected to finish
- 2017-01-11
- Last updated by the study team
- 2019-07-05
Who can join
Age: 4 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Children's Hospital & Research Center at Oakland — Oakland, California, United States
- Klinikum der Universität München — Munich, Germany
- Foundation Neurological Institute C. Besta — Milan, Italy
- Newcastle University Institute of Human Genetics — Newcastle upon Tyne, United Kingdom
Full record on ClinicalTrials.gov
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