TY  - JOUR
AU  - Klopstock, Thomas
AU  - Videnovic, Aleksandar
AU  - Bischoff, Almut Turid
AU  - Bonnet, Cecilia
AU  - Cif, Laura
AU  - Comella, Cynthia
AU  - Correa-Vela, Marta
AU  - Escolar, Maria L
AU  - Fraser, Jamie L
AU  - Gonzalez, Victoria
AU  - Hermanowicz, Neal
AU  - Jech, Robert
AU  - Jinnah, Hyder A
AU  - Kmiec, Tomasz
AU  - Lang, Anthony
AU  - Martí, Maria J
AU  - Mercimek-Andrews, Saadet
AU  - Monduy, Migvis
AU  - Nimmo, Graeme A M
AU  - Perez-Dueñas, Belen
AU  - Pfeiffer, Helle Cecilie Viekilde
AU  - Planellas, Lluis
AU  - Roze, Emmanuel
AU  - Thakur, Nivedita
AU  - Tochen, Laura
AU  - Vanegas-Arroyave, Nora
AU  - Zorzi, Giovanna
AU  - Burns, Colleen
AU  - Greblikas, Feriandas
TI  - Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
JO  - Movement disorders
VL  - 36
IS  - 6
SN  - 1531-8257
CY  - New York, NY
PB  - Wiley
M1  - DZNE-2021-00134
SP  - 1342 - 1352
PY  - 2020
N1  - ISSN 1531-8257 not unique: **3 hits**.
AB  - 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 (standard deviation) scores were 28.2 (11.4) and 27.4 (11.5) at baseline, respectively, and were 26.9 (12.5) and 24.5 (11.8) at week 24, respectively. The difference in least square mean (95
KW  - Activities of Daily Living
KW  - Double-Blind Method
KW  - Humans
KW  - Pantothenate Kinase-Associated Neurodegeneration: drug therapy
KW  - Pantothenate Kinase-Associated Neurodegeneration: genetics
KW  - Pantothenic Acid: analogs & derivatives
KW  - fosmetpantotenate (Other)
KW  - pantothenate kinase-associated neurodegeneration (Other)
KW  - randomized controlled trial (Other)
KW  - treatment (Other)
LB  - PUB:(DE-HGF)16
C2  - pmc:PMC8246547
C6  - pmid:33200489
DO  - DOI:10.1002/mds.28392
UR  - https://pub.dzne.de/record/154280
ER  -