Clinical trials in pediatric ALS: a TRICALS feasibility study

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).Obj...

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Autores: Kliest, Tessa, Rubio Pérez, Miguel Angel, van den Berg, Leonard H.
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2022
País:España
Institución:Universitat Pompeu Fabra
Repositorio:Repositorio Digital de la UPF
OAI Identifier:oai:repositori.upf.edu:10230/53818
Acceso en línea:http://hdl.handle.net/10230/53818
http://dx.doi.org/10.1080/21678421.2021.2024856
Access Level:acceso abierto
Palabra clave:Pediatric amyotrophic lateral sclerosis
Clinical trial
Clinical trials
Ethics
Pediatric investigation plan
Therapy
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spelling Clinical trials in pediatric ALS: a TRICALS feasibility studyKliest, TessaRubio Pérez, Miguel Angelvan den Berg, Leonard H.Pediatric amyotrophic lateral sclerosisClinical trialClinical trialsEthicsPediatric investigation planTherapyBackground: 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.Taylor & Francis202220222022info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfapplication/pdfhttp://hdl.handle.net/10230/53818http://dx.doi.org/10.1080/21678421.2021.2024856reponame:Repositorio Digital de la UPFinstname:Universitat Pompeu FabraInglésAmyotroph Lateral Scler Frontotemporal Degener. 2022 Nov;23(7-8):481-488© 2022 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.http://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:repositori.upf.edu:10230/538182026-06-12T07:21:37Z
dc.title.none.fl_str_mv Clinical trials in pediatric ALS: a TRICALS feasibility study
title Clinical trials in pediatric ALS: a TRICALS feasibility study
spellingShingle Clinical trials in pediatric ALS: a TRICALS feasibility study
Kliest, Tessa
Pediatric amyotrophic lateral sclerosis
Clinical trial
Clinical trials
Ethics
Pediatric investigation plan
Therapy
title_short Clinical trials in pediatric ALS: a TRICALS feasibility study
title_full Clinical trials in pediatric ALS: a TRICALS feasibility study
title_fullStr Clinical trials in pediatric ALS: a TRICALS feasibility study
title_full_unstemmed Clinical trials in pediatric ALS: a TRICALS feasibility study
title_sort Clinical trials in pediatric ALS: a TRICALS feasibility study
dc.creator.none.fl_str_mv Kliest, Tessa
Rubio Pérez, Miguel Angel
van den Berg, Leonard H.
author Kliest, Tessa
author_facet Kliest, Tessa
Rubio Pérez, Miguel Angel
van den Berg, Leonard H.
author_role author
author2 Rubio Pérez, Miguel Angel
van den Berg, Leonard H.
author2_role author
author
dc.subject.none.fl_str_mv Pediatric amyotrophic lateral sclerosis
Clinical trial
Clinical trials
Ethics
Pediatric investigation plan
Therapy
topic Pediatric amyotrophic lateral sclerosis
Clinical trial
Clinical trials
Ethics
Pediatric investigation plan
Therapy
description 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.
publishDate 2022
dc.date.none.fl_str_mv 2022
2022
2022
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
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dc.identifier.none.fl_str_mv http://hdl.handle.net/10230/53818
http://dx.doi.org/10.1080/21678421.2021.2024856
url http://hdl.handle.net/10230/53818
http://dx.doi.org/10.1080/21678421.2021.2024856
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Amyotroph Lateral Scler Frontotemporal Degener. 2022 Nov;23(7-8):481-488
dc.rights.none.fl_str_mv http://creativecommons.org/licenses/by/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv http://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
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application/pdf
dc.publisher.none.fl_str_mv Taylor & Francis
publisher.none.fl_str_mv Taylor & Francis
dc.source.none.fl_str_mv reponame:Repositorio Digital de la UPF
instname:Universitat Pompeu Fabra
instname_str Universitat Pompeu Fabra
reponame_str Repositorio Digital de la UPF
collection Repositorio Digital de la UPF
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