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

Phase 3 Completed 89 enrolled ApoPharma Food and Drug Administration (FDA) · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

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).

Timeline

Start: 2012-12-13
Primary completion: 2016-10-21
Completion: 2017-01-11

Outcome

Missed primary endpoint

registry analysis (superiority test); Deferiprone vs Placebo; p = 0.0761; Mixed Models Analysis NCT01741532 ↗

registry analysis (superiority test); Deferiprone vs Placebo; p = 0.7279; Mixed Models Analysis NCT01741532 ↗

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Deferiprone	Small molecule	15 mg/kg	Oral