Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.

Movement Disorders : Official Journal of the Movement Disorder Society
Thomas KlopstockFeriandas Greblikas

Abstract

Pantothenate kinase-associated neurodegeneration (PKAN) currently has no approved treatments. The Fosmetpantotenate Replacement Therapy pivotal trial examined whether treatment with fosmetpantotenate improves PKAN symptoms and stabilizes disease progression. This randomized, double-blind, placebo-controlled, multicenter study evaluated fosmetpantotenate, 300 mg oral dose three times daily, versus placebo over a 24-week double-blind period. Patients with pathogenic variants of PANK2, aged 6 to 65 years, with a score ≥6 on the PKAN-Activities of Daily Living (PKAN-ADL) scale were enrolled. Patients were randomized to active (fosmetpantotenate) or placebo treatment, stratified by weight and age. The primary efficacy endpoint was change from baseline at week 24 in PKAN-ADL. Between July 23, 2017, and December 18, 2018, 84 patients were randomized (fosmetpantotenate: n = 41; placebo: n = 43); all 84 patients were included in the analyses. Six patients in the placebo group discontinued treatment; two had worsening dystonia, two had poor compliance, and two died of PKAN-related complications (aspiration during feeding and disease progression with respiratory failure, respectively). Fosmetpantotenate and placebo group PKAN-ADL mean (st...Continue Reading

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Citations

Mar 26, 2021·Developmental Medicine and Child Neurology·Audrey K S SooManju A Kurian
May 4, 2021·Frontiers in Neurology·Vassilena IankovaSusanne A Schneider
May 19, 2021·Movement Disorders : Official Journal of the Movement Disorder Society·Nivedita ThakurHyder A Jinnah
Aug 28, 2021·Brain Sciences·Luca MignaniDario Finazzi

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