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000154280 1001_ $$0P:(DE-2719)2810704$$aKlopstock, Thomas$$b0$$eFirst author$$udzne
000154280 245__ $$aFosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
000154280 260__ $$aNew York, NY$$bWiley$$c2020
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000154280 520__ $$aPantothenate 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% confidence interval) at week 24 between fosmetpantotenate and placebo was -0.09 (-1.69 to 1.51; P = 0.9115). The overall incidence of treatment-emergent serious adverse events was similar in the fosmetpantotenate (8/41; 19.5%) and placebo (6/43; 14.0%) groups.Treatment with fosmetpantotenate was safe but did not improve function assessed by the PKAN-ADL in patients with PKAN. © 2020 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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000154280 650_7 $$2Other$$afosmetpantotenate
000154280 650_7 $$2Other$$apantothenate kinase-associated neurodegeneration
000154280 650_7 $$2Other$$arandomized controlled trial
000154280 650_7 $$2Other$$atreatment
000154280 650_2 $$2MeSH$$aActivities of Daily Living
000154280 650_2 $$2MeSH$$aDouble-Blind Method
000154280 650_2 $$2MeSH$$aHumans
000154280 650_2 $$2MeSH$$aPantothenate Kinase-Associated Neurodegeneration: drug therapy
000154280 650_2 $$2MeSH$$aPantothenate Kinase-Associated Neurodegeneration: genetics
000154280 650_2 $$2MeSH$$aPantothenic Acid: analogs & derivatives
000154280 7001_ $$aVidenovic, Aleksandar$$b1
000154280 7001_ $$aBischoff, Almut Turid$$b2
000154280 7001_ $$aBonnet, Cecilia$$b3
000154280 7001_ $$00000-0003-4920-4234$$aCif, Laura$$b4
000154280 7001_ $$aComella, Cynthia$$b5
000154280 7001_ $$00000-0002-6255-5197$$aCorrea-Vela, Marta$$b6
000154280 7001_ $$aEscolar, Maria L$$b7
000154280 7001_ $$aFraser, Jamie L$$b8
000154280 7001_ $$aGonzalez, Victoria$$b9
000154280 7001_ $$aHermanowicz, Neal$$b10
000154280 7001_ $$00000-0002-9732-8947$$aJech, Robert$$b11
000154280 7001_ $$aJinnah, Hyder A$$b12
000154280 7001_ $$aKmiec, Tomasz$$b13
000154280 7001_ $$aLang, Anthony$$b14
000154280 7001_ $$aMartí, Maria J$$b15
000154280 7001_ $$aMercimek-Andrews, Saadet$$b16
000154280 7001_ $$aMonduy, Migvis$$b17
000154280 7001_ $$aNimmo, Graeme A M$$b18
000154280 7001_ $$aPerez-Dueñas, Belen$$b19
000154280 7001_ $$aPfeiffer, Helle Cecilie Viekilde$$b20
000154280 7001_ $$aPlanellas, Lluis$$b21
000154280 7001_ $$aRoze, Emmanuel$$b22
000154280 7001_ $$aThakur, Nivedita$$b23
000154280 7001_ $$aTochen, Laura$$b24
000154280 7001_ $$aVanegas-Arroyave, Nora$$b25
000154280 7001_ $$aZorzi, Giovanna$$b26
000154280 7001_ $$aBurns, Colleen$$b27
000154280 7001_ $$aGreblikas, Feriandas$$b28
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