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Sökning: onr:"swepub:oai:DiVA.org:umu-192954" > Clinical trials in ...

Clinical trials in pediatric ALS: a TRICALS feasibility study

Kliest, Tessa (författare)
Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands
Van Eijk, Ruben P.A. (författare)
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
Al-Chalabi, Ammar (författare)
Department 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
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Albanese, Alberto (författare)
IRCCS Humanitas Research Hospital Rozzano, Milan, Italy
Andersen, Peter M., 1962- (författare)
Umeå universitet,Neurovetenskaper
Amador, Maria Del Mar (författare)
Département de Neurologie, Centre de référence SLA, France; Hôpital de la Pitié Salpêtrière, AP-HP, Paris, France
Bråthen, Geir (författare)
Department of Neurology, University Hospital of Trondheim, Trondheim, Norway; Department of Neuromedicine and Movement Science, Norwegian University of Science and Technology, Trondheim, Norway
Brunaud-Danel, Veronique (författare)
ALS centre, CHU, University of Lille Nord de France, Lille, France
Brylev, Lev (författare)
Bujanov Moscow City Clinical Hospital, Moscow, Russian Federation; Moscow Research and Clinical Center for Neuropsychiatry of the Healthcare Department, Moscow, Russian Federation
Camu, William (författare)
ALS Centre CHU Gui de Chauliac, University of Montpellier, Montpellier, France
De Carvalho, Mamede (författare)
Institute 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
Cereda, Cristina (författare)
Regional Newborn Screening Laboratory, Vittore Buzzi Children's Hospital-University of Milan, Italy
Cetin, Hakan (författare)
Department of Neurology, Medical University of Vienna, Vienna, Austria
Chaverri, Delia (författare)
Neurology Service, Hospital Universitario La Paz, Madrid, Spain
Chiò, Adriano (författare)
'Rita Levi Montalcini' Department of Neuroscience, ALS Centre, University of Torino, Turin, Italy; Azienda Ospedaliera Città della Salute e della Scienza, Turin, Italy
Corcia, Philippe (författare)
Centre Constitutif SLA, CHRU de Tours - Fédération des centres SLA Tours-Limoges, LitORALS, Tours, France
Couratier, Philippe (författare)
Centre Constitutif de reference SLA-Fédération Tours-Limoges, CHU de Limoges, Limoges, France
De Marchi, Fabiola (författare)
"Maggiore della Carità" University Hospital, Novara, Italy
Desnuelle, Claude (författare)
Hôpital Pasteur 2–CHU de Nice, Nice, France
Van Es, Michael A. (författare)
Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands
Esteban, JesÚs (författare)
ALS Research Lab - ALS Unit, Instituto de Investigación Sanitaria Hospital 12 de Octubre "i + 12", CIBERER, Madrid, Spain
Filosto, Massimiliano (författare)
Department of Clinical and Experimental Sciences, University of Brescia; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Brescia, Italy
GarcÍa Redondo, Alberto (författare)
ALS Research Lab - ALS Unit, Instituto de Investigación Sanitaria Hospital 12 de Octubre "i + 12", CIBERER, Madrid, Spain
Grosskreutz, Julian (författare)
Precision Neurology, Dept. of Neurology, Lübeck University Hospital, Lübeck, Germany
Hanemann, Clemens O. (författare)
University of Plymouth, Peninsula Schools of Medicine and Dentistry, Plymouth, United Kingdom
Holmøy, Trygve (författare)
Department of Neurology, Akershus University Hospital, Lørenskog, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway
Høyer, Helle (författare)
Department of Medical Genetics, Telemark Hospital, Skien, Norway
Ingre, Caroline (författare)
Department of Neurology, Karolinska University Hospital, Stockholm, Sweden; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden
Koritnik, Blaz (författare)
Institute of Clinical Neurophysiology, University Medical Centre Ljubljana, Ljubljana, Slovenia
Kuzma-Kozakiewicz, Magdalena (författare)
Department of Neurology, Medical University of Warsaw, Warsaw, Poland
Lambert, Thomas (författare)
Department of Neurology, Royal Stoke University Hospital, Stoke, United Kingdom
Leigh, Peter N. (författare)
Department of Neuroscience, Brighton and Sussex Medical School, Trafford Centre for Biomedical Research, University of Sussex, Brighton, United Kingdom
Lunetta, Christian (författare)
NEMO Clinical Center, Serena Onlus Foundation, Milan, Italy; NEMO LAB, Milan, Italy
Mandrioli, Jessica (författare)
Department 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
Mcdermott, Christopher J. (författare)
Department of Neuroscience, University of Sheffield, Sheffield Institute for Translational Neuroscience, Sheffield, United Kingdom
Meyer, Thomas (författare)
ALS Outpatient Department, Charité–Universitatsmedizin Berlin, Berlin, Germany
Mora, Jesus S. (författare)
ALS Unit/Neurology, Hospital San Rafael, Madrid, Spain
Petri, Susanne (författare)
Department of Neurology, Hannover Medical School, Hannover, Germany
Povedano, Mónica (författare)
Functional Unit of Amyotrophic Lateral Sclerosis (UFELA), Service of Neurology, Bellvitge University Hospital, Hospitalet de Llobregat, Spain
Reviers, Evy (författare)
European Organization for Professionals and Patients with ALS (EUpALS) & ALS Liga Belgium, Leuven, Belgium
Riva, Nilo (författare)
Department of Neurology, Experimental Neuropathology Unit, Institute of Experimental Neurology, Division of Neuroscience, San Raffaele Scientific Institute, Milan, Italy
Roes, Kit C.B. (författare)
Department of Health Evidence, Section Biostatistics, Radboud University Medical Centre Nijmegen, Nijmegen, Netherlands
Rubio, Miguel Á. (författare)
Neuromuscular Unit, Department of Neurology, Hospital del Mar, Barcelona, Spain; Instituto Hospital del Mar de Investivaciones Médicas (IMIM), Barcelona, Spain
Salachas, François (författare)
Département de Neurologie, Centre de référence SLA, France; Hôpital de la Pitié Salpêtrière, AP-HP, Paris, France
Sarafov, Stayko (författare)
Clinic of General Neurology, Medical University Sofia, University Hospital Alexandrovska, Sofia, Bulgaria
Sorarù, Gianni (författare)
Department of Neurosciences, University of Padova, Padova, Italy
Stevic, Zorica (författare)
Clinic of Neurology, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia
Svenstrup, Kirsten (författare)
Department of Neurology, Bispebjerg-Frederiksberg Hospital and Rigshospitalet, University Hospital of Copenhagen, Denmark
Møller, Anette Torvin (författare)
Department of Neurology, Aarhus University Hospital, Aarhus, Denmark
Turner, Martin R. (författare)
Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom
Van Damme, Philip (författare)
Department 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
Van Leeuwen, Lucie A.G. (författare)
Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands
Varona, Luis (författare)
Department of Neurology, Basurto University Hospital, Vizcaya, Spain
VÁzquez Costa, Juan F. (författare)
ALS Unit and Neuromuscular Disease Unit, Department of Neurology, Hospital La Fe, Valencia, Spain
Weber, Markus (författare)
Neuromoscular Disease Unit/ALS Clinic, Cantonal Hospital St. Gallen, St. Gallen, Switzerland
Hardiman, Orla (författare)
Academic Unit of Neurology Trinity College Dublin Ireland, Dublin, Ireland
Van Den Berg, Leonard H. (författare)
Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands
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 (creator_code:org_t)
2022-02-16
2022
Engelska.
Ingår i: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. - : Taylor & Francis Group. - 2167-8421 .- 2167-9223. ; 23:7-8, s. 481-488
  • Tidskriftsartikel (refereegranskat)
Abstract Ämnesord
Stäng  
  • 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.

Ämnesord

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Neurologi (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Neurology (hsv//eng)
HUMANIORA  -- Filosofi, etik och religion -- Etik (hsv//swe)
HUMANITIES  -- Philosophy, Ethics and Religion -- Ethics (hsv//eng)
MEDICIN OCH HÄLSOVETENSKAP  -- Medicinska och farmaceutiska grundvetenskaper -- Neurovetenskaper (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Basic Medicine -- Neurosciences (hsv//eng)

Nyckelord

clinical trial
clinical trials
ethics
Pediatric amyotrophic lateral sclerosis
pediatric investigation plan
therapy

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