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  • Kliest, TessaDepartment of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands (author)

Clinical trials in pediatric ALS: a TRICALS feasibility study

  • Article/chapterEnglish2022

Publisher, publication year, extent ...

  • 2022-02-16
  • Taylor & Francis Group,2022
  • electronicrdacarrier

Numbers

  • LIBRIS-ID:oai:DiVA.org:umu-192954
  • https://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-192954URI
  • https://doi.org/10.1080/21678421.2021.2024856DOI
  • http://kipublications.ki.se/Default.aspx?queryparsed=id:148845760URI

Supplementary language notes

  • Language:English
  • Summary in:English

Part of subdatabase

Classification

  • Subject category:ref swepub-contenttype
  • Subject category:art swepub-publicationtype

Notes

  • 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 (<18 years) with ALS in Europe.Methods: The EMA database was searched for submitted PIPs in ALS. A questionnaire was sent to 58 European ALS centers to collect the prevalence of pediatric ALS during the past ten years, the recruitment potential for future pediatric trials, and opinions of ALS experts concerning a waiver for ALS.Results: Four PIPs were identified; two were waived and two are planned for the future. In total, 49 (84.5%) centers responded to the questionnaire. The diagnosis of 44,858 patients with ALS was reported by 46 sites; 39 of the patients had an onset < 18 years (prevalence of 0.008 cases per 100,000 or 0.087% of all diagnosed patients). The estimated recruitment potential (47 sites) was 26 pediatric patients within five years. A majority of ALS experts (75.5%) recommend a waiver should apply for ALS due to the low prevalence of pediatric ALS.Conclusions: ALS with an onset before 18 years is extremely rare and may be a distinct entity from adult ALS. Conducting studies on the effect of disease-modifying therapy in pediatric ALS may involve lengthy recruitment periods, high costs, ethical/legal implications, challenges in trial design and limited information.

Subject headings and genre

Added entries (persons, corporate bodies, meetings, titles ...)

  • Van Eijk, Ruben P.A.Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands; Biostatistics & Research Support, Julius Centre for Health Sciences and Primary Care, University Medical Centre Utrecht, Utrecht, Netherlands (author)
  • Al-Chalabi, AmmarDepartment of Basic and Clinical Neuroscience, King's College London, Maurice Wohl Clinical Neuroscience Institute, London, United Kingdom; Department of Neurology, King’s College Hospital, London, United Kingdom (author)
  • Albanese, AlbertoIRCCS Humanitas Research Hospital Rozzano, Milan, Italy (author)
  • Andersen, Peter M.,1962-Umeå universitet,Neurovetenskaper(Swepub:umu)pean0001 (author)
  • Amador, Maria Del MarDépartement de Neurologie, Centre de référence SLA, France; Hôpital de la Pitié Salpêtrière, AP-HP, Paris, France (author)
  • Bråthen, GeirDepartment of Neurology, University Hospital of Trondheim, Trondheim, Norway; Department of Neuromedicine and Movement Science, Norwegian University of Science and Technology, Trondheim, Norway (author)
  • Brunaud-Danel, VeroniqueALS centre, CHU, University of Lille Nord de France, Lille, France (author)
  • Brylev, LevBujanov Moscow City Clinical Hospital, Moscow, Russian Federation; Moscow Research and Clinical Center for Neuropsychiatry of the Healthcare Department, Moscow, Russian Federation (author)
  • Camu, WilliamALS Centre CHU Gui de Chauliac, University of Montpellier, Montpellier, France (author)
  • De Carvalho, MamedeInstitute of Physiology-Instituto de Medicina Molecular, Faculty of Medicine, University of Lisbon, Lisbon, Portugal; Department of Neurosciences and Mental Health, H Santa Maria-CHLN, Lisbon, Portugal (author)
  • Cereda, CristinaKarolinska Institutet (author)
  • Cetin, HakanDepartment of Neurology, Medical University of Vienna, Vienna, Austria (author)
  • Chaverri, DeliaNeurology Service, Hospital Universitario La Paz, Madrid, Spain (author)
  • Chiò, Adriano'Rita Levi Montalcini' Department of Neuroscience, ALS Centre, University of Torino, Turin, Italy; Azienda Ospedaliera Città della Salute e della Scienza, Turin, Italy (author)
  • Corcia, PhilippeCentre Constitutif SLA, CHRU de Tours - Fédération des centres SLA Tours-Limoges, LitORALS, Tours, France (author)
  • Couratier, PhilippeCentre Constitutif de reference SLA-Fédération Tours-Limoges, CHU de Limoges, Limoges, France (author)
  • De Marchi, Fabiola"Maggiore della Carità" University Hospital, Novara, Italy (author)
  • Desnuelle, ClaudeHôpital Pasteur 2–CHU de Nice, Nice, France (author)
  • Van Es, Michael A.Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands (author)
  • Esteban, JesÚsALS Research Lab - ALS Unit, Instituto de Investigación Sanitaria Hospital 12 de Octubre "i + 12", CIBERER, Madrid, Spain (author)
  • Filosto, MassimilianoDepartment of Clinical and Experimental Sciences, University of Brescia; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Brescia, Italy (author)
  • GarcÍa Redondo, AlbertoALS Research Lab - ALS Unit, Instituto de Investigación Sanitaria Hospital 12 de Octubre "i + 12", CIBERER, Madrid, Spain (author)
  • Grosskreutz, JulianPrecision Neurology, Dept. of Neurology, Lübeck University Hospital, Lübeck, Germany (author)
  • Hanemann, Clemens O.University of Plymouth, Peninsula Schools of Medicine and Dentistry, Plymouth, United Kingdom (author)
  • Holmøy, TrygveDepartment of Neurology, Akershus University Hospital, Lørenskog, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway (author)
  • Høyer, HelleDepartment of Medical Genetics, Telemark Hospital, Skien, Norway (author)
  • Ingre, CarolineDepartment of Neurology, Karolinska University Hospital, Stockholm, Sweden; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden (author)
  • Koritnik, BlazInstitute of Clinical Neurophysiology, University Medical Centre Ljubljana, Ljubljana, Slovenia (author)
  • Kuzma-Kozakiewicz, MagdalenaDepartment of Neurology, Medical University of Warsaw, Warsaw, Poland (author)
  • Lambert, ThomasDepartment of Neurology, Royal Stoke University Hospital, Stoke, United Kingdom (author)
  • Leigh, Peter N.Department of Neuroscience, Brighton and Sussex Medical School, Trafford Centre for Biomedical Research, University of Sussex, Brighton, United Kingdom (author)
  • Lunetta, ChristianNEMO Clinical Center, Serena Onlus Foundation, Milan, Italy; NEMO LAB, Milan, Italy (author)
  • Mandrioli, JessicaDepartment of Biomedical, Metabolic and Neural Sciences, Center for Neuroscience and Neurotechnology, University of Modena and Reggio Emilia, Modena, Italy; Department of Neuroscience, St. Agostino Estense Hospital, Azienda Ospedaliero Universitaria di Modena, Modena, Italy (author)
  • Mcdermott, Christopher J.Department of Neuroscience, University of Sheffield, Sheffield Institute for Translational Neuroscience, Sheffield, United Kingdom (author)
  • Meyer, ThomasALS Outpatient Department, Charité–Universitatsmedizin Berlin, Berlin, Germany (author)
  • Mora, Jesus S.ALS Unit/Neurology, Hospital San Rafael, Madrid, Spain (author)
  • Petri, SusanneDepartment of Neurology, Hannover Medical School, Hannover, Germany (author)
  • Povedano, MónicaFunctional Unit of Amyotrophic Lateral Sclerosis (UFELA), Service of Neurology, Bellvitge University Hospital, Hospitalet de Llobregat, Spain (author)
  • Reviers, EvyEuropean Organization for Professionals and Patients with ALS (EUpALS) & ALS Liga Belgium, Leuven, Belgium (author)
  • Riva, NiloDepartment of Neurology, Experimental Neuropathology Unit, Institute of Experimental Neurology, Division of Neuroscience, San Raffaele Scientific Institute, Milan, Italy (author)
  • Roes, Kit C.B.Department of Health Evidence, Section Biostatistics, Radboud University Medical Centre Nijmegen, Nijmegen, Netherlands (author)
  • Rubio, Miguel Á.Neuromuscular Unit, Department of Neurology, Hospital del Mar, Barcelona, Spain; Instituto Hospital del Mar de Investivaciones Médicas (IMIM), Barcelona, Spain (author)
  • Salachas, FrançoisDépartement de Neurologie, Centre de référence SLA, France; Hôpital de la Pitié Salpêtrière, AP-HP, Paris, France (author)
  • Sarafov, StaykoClinic of General Neurology, Medical University Sofia, University Hospital Alexandrovska, Sofia, Bulgaria (author)
  • Sorarù, GianniDepartment of Neurosciences, University of Padova, Padova, Italy (author)
  • Stevic, ZoricaClinic of Neurology, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia (author)
  • Svenstrup, KirstenDepartment of Neurology, Bispebjerg-Frederiksberg Hospital and Rigshospitalet, University Hospital of Copenhagen, Denmark (author)
  • Møller, Anette TorvinDepartment of Neurology, Aarhus University Hospital, Aarhus, Denmark (author)
  • Turner, Martin R.Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom (author)
  • Van Damme, PhilipDepartment of Neurosciences, Laboratory for Neurobiology, KU Leuven and Centre for Brain & Disease Research, VIB, Leuven Brain Institute, Leuven, Belgium; Department of Neurology, University Hospitals Leuven, Leuven, Belgium (author)
  • Van Leeuwen, Lucie A.G.Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands (author)
  • Varona, LuisDepartment of Neurology, Basurto University Hospital, Vizcaya, Spain (author)
  • VÁzquez Costa, Juan F.ALS Unit and Neuromuscular Disease Unit, Department of Neurology, Hospital La Fe, Valencia, Spain (author)
  • Weber, MarkusNeuromoscular Disease Unit/ALS Clinic, Cantonal Hospital St. Gallen, St. Gallen, Switzerland (author)
  • Hardiman, OrlaAcademic Unit of Neurology Trinity College Dublin Ireland, Dublin, Ireland (author)
  • Van Den Berg, Leonard H.Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands (author)
  • Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, NetherlandsDepartment of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands; Biostatistics & Research Support, Julius Centre for Health Sciences and Primary Care, University Medical Centre Utrecht, Utrecht, Netherlands (creator_code:org_t)

Related titles

  • In:Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration: Taylor & Francis Group23:7-8, s. 481-4882167-84212167-9223

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