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Clinical trials in pediatric ALS: a TRICALS feasibility study

Authors :
Tessa Kliest
Ruben P.A. Van Eijk
Ammar Al-Chalabi
Alberto Albanese
Peter M. Andersen
Maria Del Mar Amador
Geir BrÅthen
Veronique Brunaud-Danel
Lev Brylev
William Camu
Mamede De Carvalho
Cristina Cereda
Hakan Cetin
Delia Chaverri
Adriano Chiò
Philippe Corcia
Philippe Couratier
Fabiola De Marchi
Claude Desnuelle
Michael A. Van Es
JesÚs Esteban
Massimiliano Filosto
Alberto GarcÍa Redondo
Julian Grosskreutz
Clemens O. Hanemann
Trygve HolmØy
Helle HØyer
Caroline Ingre
Blaz Koritnik
Magdalena Kuzma-Kozakiewicz
Thomas Lambert
Peter N. Leigh
Christian Lunetta
Jessica Mandrioli
Christopher J. Mcdermott
Thomas Meyer
Jesus S. Mora
Susanne Petri
MÓnica Povedano
Evy Reviers
Nilo Riva
Kit C.B. Roes
Miguel Á. Rubio
FranÇois Salachas
Stayko Sarafov
Gianni SorarÙ
Zorica Stevic
Kirsten Svenstrup
Anette Torvin MØller
Martin R. Turner
Philip Van Damme
Lucie A.G. Van Leeuwen
Luis Varona
Juan F. VÁzquez Costa
Markus Weber
Orla Hardiman
Leonard H. Van Den Berg
Department of Neurology, Rudolf Magnus Institute of Neuroscience, University Medical Centre Utrecht
Maurice Wohl Clinical Neuroscience Institut
King‘s College London
King's College Hospital (KCH)
Department Neurological Sciences, Milan University, IRCCS Humanitas Clinical Institute, Rozzano, M
Umeå University
CHU Pitié-Salpêtrière [AP-HP]
Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)
Université de Lille
Ministry of Healthcare of Russian Federation
Department of Neurology, Montpellier CHU, Gui De Chauliac Hospital, Montpellier
Université de Lisbonne
Medizinische Universität Wien = Medical University of Vienna
Hospital Universitario La Paz
Università degli studi di Torino = University of Turin (UNITO)
Centre de compétence de la Sclérose Latérale Amyotrophique [CHRU Tours] (SLA CHRU Tours)
Centre Hospitalier Régional Universitaire de Tours (CHRU Tours)
Epidémiologie des Maladies Chroniques en zone tropicale (EpiMaCT)
CHU Limoges-Institut d'Epidémiologie Neurologique et de Neurologie Tropicale-Institut National de la Santé et de la Recherche Médicale (INSERM)-OmégaHealth (ΩHealth)
Université de Limoges (UNILIM)-Université de Limoges (UNILIM)
Service de Neurologie [CHU Limoges]
CHU Limoges
Centre référent Sclérose Latérale Amyotrophique et autres maladies du motoneurone [CHU Limoges] (SLA CHU Limoges)
Neurology Clinic, University Hospital 'Maggiore della Carità', Novara
Hôpital Pasteur [Nice] (CHU)
Centro de Diagnóstico de Enfermedades Moleculares, Dpto. Biol. Mol. Centro de Biología Molecular-SO UAM-CSIC. Universidad Autónoma Madrid, Campus Cantoblanco
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Car
University of Brescia
Lubeck University Hospital
Peninsula Schools of Medicine and Dentistry
Plymouth University
University of Oslo (UiO)
Akershus University Hospital [Lørenskog]
Telemark Hospital Trust [Skien, Norway]
Karolinska Institutet, Department of Neurology
Karolinska University Hospital [Stockholm]
University Medical Centre Ljubljana [Ljubljana, Slovenia] (UMCL)
Departement de Neurology (MEDICAL UNIVERSITY OF WARSAW)
Medical University of Warsaw - Poland
University Hospitals of North Midlands NHS Trust, Royal Stoke Hospital, Stoke-on-Trent, United Kingdom.
University of Sussex
University of Modena and Reggio Emilia, Department of Biomedical Sciences
Department of Neuroscience, Academic Neurology Unit, University of Sheffield
Sant Rafael Hospital
Department of Neurology, Hannover Medical School
Hospital Universitari de Bellvitge, L'Hospitalet de Llobregat, University of Barcelona
Leuven University Hospitals
Division of Neuroscience, San Raffaele Scientific Institute
San Raffaele Scientific Institute, Milan, Italy
Radboud University Medical Center [Nijmegen]
IMIM-Hospital del Mar
Generalitat de Catalunya
Medical University of Sofia [Bulgarie]
Université de Padoue
Institute of Neurology, Clinical Center of Serbia, University of Belgrade, Belgrade
University of Belgrade [Belgrade]
University of Copenhagen = Københavns Universitet (UCPH)
Aarhus University Hospital
University of Oxford, Nuffield Department of Clinical Neurosciences, Oxford, UK
University Hospitals Leuven [Leuven]
Servicio de Neurología, Hospital de Cruces, Barakaldo, Vizcaya
Neurology Service, Hospital Universitario La Fe, Valencia
Cantonal Hospital St Gallen (KSSG)
Department of Neurology, The Adelaide and Meath Hospital, Trinity College Dublin
Source :
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 2022, pp.1-8. ⟨10.1080/21678421.2021.2024856⟩, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 23, 481-488, r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe, instname, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 23, 7-8, pp. 481-488, Kliest, T, Van Eijk, R P A, Al-Chalabi, A, Albanese, A, Andersen, P M, Amador, M D M, BrÅthen, G, Brunaud-Danel, V, Brylev, L, Camu, W, De Carvalho, M, Cereda, C, Cetin, H, Chaverri, D, Chiò, A, Corcia, P, Couratier, P, De Marchi, F, Desnuelle, C, Van Es, M A, Esteban, J, Filosto, M, GarcÍa Redondo, A, Grosskreutz, J, Hanemann, C O, HolmØy, T, HØyer, H, Ingre, C, Koritnik, B, Kuzma-Kozakiewicz, M, Lambert, T, Leigh, P N, Lunetta, C, Mandrioli, J, Mcdermott, C J, Meyer, T, Mora, J S, Petri, S, Povedano, MÓ, Reviers, E, Riva, N, Roes, K C B, Rubio, M, Salachas, F, Sarafov, S, SorarÙ, G, Stevic, Z, Svenstrup, K, MØller, A T, Turner, M R, Van Damme, P, Van Leeuwen, L A G, Varona, L, VÁzquez Costa, J F, Weber, M, Hardiman, O & Van Den Berg, L H 2022, ' Clinical trials in pediatric ALS : a TRICALS feasibility study ', Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol. 23, no. 7-8, pp. 481-488 . https://doi.org/10.1080/21678421.2021.2024856
Publication Year :
2022
Publisher :
HAL CCSD, 2022.

Abstract

Background: Pediatric investigation plans (PIPs) describe how adult drugs can be studied in children. In 2015, PIPs for Amyotrophic Lateral Sclerosis (ALS) became mandatory for European marketing-authorization of adult treatments, unless a waiver is granted by the European Medicines Agency (EMA).Objective: To assess the feasibility of clinical studies on the effect of therapy in children (

Subjects

Subjects :
Adult
Databases, Factual
Neurologi
GENETICS
Clinical Neurology
AMYOTROPHIC-LATERAL-SCLEROSIS
Pediatric amyotrophic lateral sclerosis
pediatric investigation plan
Databases
Prevalence
Humans
clinical trial
clinical trials
ethics
therapy
Child
Feasibility Studies
Europe
Amyotrophic Lateral Sclerosis
Factual
Science & Technology
Disorders of movement Donders Center for Medical Neuroscience [Radboudumc 3]
Neurology
[SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie
Neurology (clinical)
Neurosciences & Neurology
Life Sciences & Biomedicine

Details

Language :
English
ISSN :
21678421
Database :
OpenAIRE
Journal :
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 2022, pp.1-8. ⟨10.1080/21678421.2021.2024856⟩, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 23, 481-488, r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe, instname, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 23, 7-8, pp. 481-488, Kliest, T, Van Eijk, R P A, Al-Chalabi, A, Albanese, A, Andersen, P M, Amador, M D M, BrÅthen, G, Brunaud-Danel, V, Brylev, L, Camu, W, De Carvalho, M, Cereda, C, Cetin, H, Chaverri, D, Chiò, A, Corcia, P, Couratier, P, De Marchi, F, Desnuelle, C, Van Es, M A, Esteban, J, Filosto, M, GarcÍa Redondo, A, Grosskreutz, J, Hanemann, C O, HolmØy, T, HØyer, H, Ingre, C, Koritnik, B, Kuzma-Kozakiewicz, M, Lambert, T, Leigh, P N, Lunetta, C, Mandrioli, J, Mcdermott, C J, Meyer, T, Mora, J S, Petri, S, Povedano, MÓ, Reviers, E, Riva, N, Roes, K C B, Rubio, M, Salachas, F, Sarafov, S, SorarÙ, G, Stevic, Z, Svenstrup, K, MØller, A T, Turner, M R, Van Damme, P, Van Leeuwen, L A G, Varona, L, VÁzquez Costa, J F, Weber, M, Hardiman, O & Van Den Berg, L H 2022, ' Clinical trials in pediatric ALS : a TRICALS feasibility study ', Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol. 23, no. 7-8, pp. 481-488 . https://doi.org/10.1080/21678421.2021.2024856
Accession number :
edsair.doi.dedup.....31fe6c6cded34ce9af67049e3ef23c06

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Authors Abstract Subjects Details