Pantothenate Kinase-Associated Neurodegeneration Clinical Trial
Official title:
A Randomized, Double-blind, Placebo-controlled Trial of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
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).
This is a multi-center, double-blind, randomized, placebo-controlled, 18-month study in patients with PKAN aged 4 years and older. Participants are randomized in a 2:1 ratio to receive either deferiprone oral solution or placebo, twice a day for 18 months. Efficacy assessments, an MRI scan to measure iron levels in the globus pallidus, pharmacokinetic evaluations, and safety assessments are conducted at specified time points. Following completion of the trial, eligible patients are invited to enroll in an 18-month extension study, TIRCON2012V1-EXT, in which all participants receive deferiprone. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
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Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
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Phase 3 | |
| Terminated |
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Phase 3 | |
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N/A | |
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