Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration

Fosmetpantotenate; Randomized controlled trial

Detalles Bibliográficos
Autores: Videnovic, Aleksandar, Bischoff, Almut Turid, Bonnet, Cecilia, Cif, Laura, Comella, Cynthia, Correa Vela, Marta, Perez Dueñas, Belen, Klopstock, Thomas
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2021
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:11351/7782
Acceso en línea:https://hdl.handle.net/11351/7782
http://hdl.handle.net/11351/7782
Access Level:acceso abierto
Palabra clave:Neurologia pediàtrica
Vitamines B
DISEASES::Nervous System Diseases::Central Nervous System Diseases::Brain Diseases::Basal Ganglia Diseases::Pantothenate Kinase-Associated Neurodegeneration
Other subheadings::Other subheadings::Other subheadings::/drug therapy
ENFERMEDADES::enfermedades del sistema nervioso::enfermedades del sistema nervioso central::enfermedades cerebrales::enfermedades de los ganglios basales::neurodegeneración asociada a pantotenato cinasa
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
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oai_identifier_str oai:recercat.cat:11351/7782
network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
title Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
spellingShingle Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
Videnovic, Aleksandar
Neurologia pediàtrica
Vitamines B
DISEASES::Nervous System Diseases::Central Nervous System Diseases::Brain Diseases::Basal Ganglia Diseases::Pantothenate Kinase-Associated Neurodegeneration
Other subheadings::Other subheadings::Other subheadings::/drug therapy
ENFERMEDADES::enfermedades del sistema nervioso::enfermedades del sistema nervioso central::enfermedades cerebrales::enfermedades de los ganglios basales::neurodegeneración asociada a pantotenato cinasa
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
title_short Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
title_full Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
title_fullStr Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
title_full_unstemmed Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
title_sort Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated Neurodegeneration
dc.creator.none.fl_str_mv Videnovic, Aleksandar
Bischoff, Almut Turid
Bonnet, Cecilia
Cif, Laura
Comella, Cynthia
Correa Vela, Marta
Perez Dueñas, Belen
Klopstock, Thomas
author Videnovic, Aleksandar
author_facet Videnovic, Aleksandar
Bischoff, Almut Turid
Bonnet, Cecilia
Cif, Laura
Comella, Cynthia
Correa Vela, Marta
Perez Dueñas, Belen
Klopstock, Thomas
author_role author
author2 Bischoff, Almut Turid
Bonnet, Cecilia
Cif, Laura
Comella, Cynthia
Correa Vela, Marta
Perez Dueñas, Belen
Klopstock, Thomas
author2_role author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Institut Català de la Salut
[Klopstock T] Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, Munich, Germany. German Center for Neurodegenerative Diseases (DZNE), Munich, Munich, Germany. Munich Cluster for Systems Neurology (SyNergy), Munich, Munich, Germany. [Videnovic A] Department of Neurology, Massachusetts General Hospital/Harvard Medical School, Boston, Massachusetts, USA. [Bischoff AT] Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, Munich, Germany. [Bonnet C] Department of Neurology, Sorbonne University, AP-HP Salpêtrière Hospital, Paris, France. [Cif L] Department of Neurosurgery, CHRU de Montpellier, Gui de Chauliac Hospital, Montpellier, France. [Comella C] Department of Neurosurgery and Neurological Sciences, Rush University Medical Center, Chicago, Illinois, USA. [Correa-Vela M, Perez-Dueñas B] Servei de Neurologia Pediàtrica, Vall d’Hebron Hospital Universitari, Barcelona, Spain
Vall d'Hebron Barcelona Hospital Campus
dc.subject.none.fl_str_mv Neurologia pediàtrica
Vitamines B
DISEASES::Nervous System Diseases::Central Nervous System Diseases::Brain Diseases::Basal Ganglia Diseases::Pantothenate Kinase-Associated Neurodegeneration
Other subheadings::Other subheadings::Other subheadings::/drug therapy
ENFERMEDADES::enfermedades del sistema nervioso::enfermedades del sistema nervioso central::enfermedades cerebrales::enfermedades de los ganglios basales::neurodegeneración asociada a pantotenato cinasa
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
topic Neurologia pediàtrica
Vitamines B
DISEASES::Nervous System Diseases::Central Nervous System Diseases::Brain Diseases::Basal Ganglia Diseases::Pantothenate Kinase-Associated Neurodegeneration
Other subheadings::Other subheadings::Other subheadings::/drug therapy
ENFERMEDADES::enfermedades del sistema nervioso::enfermedades del sistema nervioso central::enfermedades cerebrales::enfermedades de los ganglios basales::neurodegeneración asociada a pantotenato cinasa
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
description Fosmetpantotenate; Randomized controlled trial
publishDate 2021
dc.date.none.fl_str_mv 2021
2022
2022
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/11351/7782
http://hdl.handle.net/11351/7782
url https://hdl.handle.net/11351/7782
http://hdl.handle.net/11351/7782
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Movement Disorders;36(6)
https://doi.org/10.1002/mds.28392
dc.rights.none.fl_str_mv Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Wiley
publisher.none.fl_str_mv Wiley
dc.source.none.fl_str_mv Scientia
reponame:Recercat. Dipósit de la Recerca de Catalunya
instname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
instname_str Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
reponame_str Recercat. Dipósit de la Recerca de Catalunya
collection Recercat. Dipósit de la Recerca de Catalunya
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869422208481230848
spelling Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase–Associated NeurodegenerationVidenovic, AleksandarBischoff, Almut TuridBonnet, CeciliaCif, LauraComella, CynthiaCorrea Vela, MartaPerez Dueñas, BelenKlopstock, ThomasNeurologia pediàtricaVitamines BDISEASES::Nervous System Diseases::Central Nervous System Diseases::Brain Diseases::Basal Ganglia Diseases::Pantothenate Kinase-Associated NeurodegenerationOther subheadings::Other subheadings::Other subheadings::/drug therapyENFERMEDADES::enfermedades del sistema nervioso::enfermedades del sistema nervioso central::enfermedades cerebrales::enfermedades de los ganglios basales::neurodegeneración asociada a pantotenato cinasaOtros calificadores::Otros calificadores::Otros calificadores::/farmacoterapiaFosmetpantotenate; Randomized controlled trialFosmetpantotenato; Ensayo controlado aleatorizadoFosmetpantotenat; Assaig controlat aleatoritzatBackground Pantothenate kinase–associated neurodegeneration (PKAN) currently has no approved treatments. Objectives The Fosmetpantotenate Replacement Therapy pivotal trial examined whether treatment with fosmetpantotenate improves PKAN symptoms and stabilizes disease progression. Methods 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. Results 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. Conclusions Treatment with fosmetpantotenate was safe but did not improve function assessed by the PKAN-ADL in patients with PKAN.The FORT trial was supported by Retrophin, Inc.WileyInstitut Català de la Salut[Klopstock T] Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, Munich, Germany. German Center for Neurodegenerative Diseases (DZNE), Munich, Munich, Germany. Munich Cluster for Systems Neurology (SyNergy), Munich, Munich, Germany. [Videnovic A] Department of Neurology, Massachusetts General Hospital/Harvard Medical School, Boston, Massachusetts, USA. [Bischoff AT] Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, Munich, Germany. [Bonnet C] Department of Neurology, Sorbonne University, AP-HP Salpêtrière Hospital, Paris, France. [Cif L] Department of Neurosurgery, CHRU de Montpellier, Gui de Chauliac Hospital, Montpellier, France. [Comella C] Department of Neurosurgery and Neurological Sciences, Rush University Medical Center, Chicago, Illinois, USA. [Correa-Vela M, Perez-Dueñas B] Servei de Neurologia Pediàtrica, Vall d’Hebron Hospital Universitari, Barcelona, SpainVall d'Hebron Barcelona Hospital Campus202220222021info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/11351/7782http://hdl.handle.net/11351/7782Scientiareponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésMovement Disorders;36(6)https://doi.org/10.1002/mds.28392Attribution-NonCommercial 4.0 Internationalhttp://creativecommons.org/licenses/by-nc/4.0/info:eu-repo/semantics/openAccessoai:recercat.cat:11351/77822026-05-29T05:05:01Z
score 15,81155

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