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dc.contributor.authorKliest, T
dc.contributor.authorVan Eijk, RPA
dc.contributor.authorAl-Chalabi, A
dc.contributor.authorAlbanese, A
dc.contributor.authorAndersen, PM
dc.contributor.authorAmador, MDM
dc.contributor.authorBrÅthen, G
dc.contributor.authorBrunaud-Danel, V
dc.contributor.authorBrylev, L
dc.contributor.authorCamu, W
dc.contributor.authorDe Carvalho, M
dc.contributor.authorCereda, C
dc.contributor.authorCetin, H
dc.contributor.authorChaverri, D
dc.contributor.authorChiò, A
dc.contributor.authorCorcia, P
dc.contributor.authorCouratier, P
dc.contributor.authorDe Marchi, F
dc.contributor.authorDesnuelle, C
dc.contributor.authorVan Es, MA
dc.contributor.authorEsteban, J
dc.contributor.authorFilosto, M
dc.contributor.authorGarcÍa Redondo, A
dc.contributor.authorGrosskreutz, J
dc.contributor.authorHanemann, Clemens Oliver
dc.contributor.authorHolmØy, T
dc.contributor.authorHØyer, H
dc.contributor.authorIngre, C
dc.contributor.authorKoritnik, B
dc.contributor.authorKuzma-Kozakiewicz, M
dc.contributor.authorLambert, T
dc.contributor.authorLeigh, PN
dc.contributor.authorLunetta, C
dc.contributor.authorMandrioli, J
dc.contributor.authorMcdermott, CJ
dc.contributor.authorMeyer, T
dc.contributor.authorMora, JS
dc.contributor.authorPetri, S
dc.contributor.authorPovedano, M
dc.contributor.authorReviers, E
dc.contributor.authorRiva, N
dc.contributor.authorRoes, KCB
dc.contributor.authorRubio, MÁ
dc.contributor.authorSalachas, F
dc.contributor.authorSarafov, S
dc.contributor.authorSorarÙ, G
dc.contributor.authorStevic, Z
dc.contributor.authorSvenstrup, K
dc.contributor.authorMØller, AT
dc.contributor.authorTurner, MR
dc.contributor.authorVan Damme, P
dc.contributor.authorVan Leeuwen, LAG
dc.contributor.authorVarona, L
dc.contributor.authorVÁzquez Costa, JF
dc.contributor.authorWeber, M
dc.contributor.authorHardiman, O
dc.contributor.authorVan Den Berg, LH

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.

dc.publisherTaylor & Francis
dc.subjectPediatric amyotrophic lateral sclerosis
dc.subjectclinical trial
dc.subjectpediatric investigation plan
dc.subjectclinical trials
dc.titleClinical trials in pediatric ALS: a TRICALS feasibility study
dc.typeJournal Article
dc.typeResearch Support, Non-U.S. Gov't
plymouth.journalAmyotrophic lateral sclerosis & frontotemporal degeneration
plymouth.organisational-group/Plymouth/Faculty of Health
plymouth.organisational-group/Plymouth/Faculty of Health/Peninsula Medical School
plymouth.organisational-group/Plymouth/REF 2021 Researchers by UoA
plymouth.organisational-group/Plymouth/REF 2021 Researchers by UoA/UoA01 Clinical Medicine
plymouth.organisational-group/Plymouth/Research Groups
plymouth.organisational-group/Plymouth/Research Groups/Institute of Translational and Stratified Medicine (ITSMED)
plymouth.organisational-group/Plymouth/Research Groups/Institute of Translational and Stratified Medicine (ITSMED)/CBR
plymouth.organisational-group/Plymouth/Users by role
plymouth.organisational-group/Plymouth/Users by role/Academics
plymouth.organisational-group/Plymouth/Users by role/Researchers in ResearchFish submission
dc.rights.embargoperiodNot known
rioxxterms.typeJournal Article/Review

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