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

PHASE3CompletedINTERVENTIONAL
Enrollment

89

Participants

Timeline

Start Date

December 13, 2012

Primary Completion Date

October 21, 2016

Study Completion Date

January 11, 2017

Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
DRUG

Deferiprone oral solution

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.

DRUG

Placebo

A deferiprone matching placebo oral solution will be given twice daily for 18 months.

Trial Locations (4)

20133

Foundation Neurological Institute C. Besta, Milan

80336

Klinikum der Universität München, Munich

94609

Children's Hospital & Research Center at Oakland, Oakland

NE1 3BZ

Newcastle University Institute of Human Genetics, Newcastle upon Tyne

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

Food and Drug Administration (FDA)

FED

lead

ApoPharma

INDUSTRY

NCT01741532 - Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN) | Biotech Hunter | Biotech Hunter