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Clinical trials in pediatric ALS: a TRICALS feasibility study

Tessa Kliest 1 Ruben P.A. van Eijk 1 Ammar Al-Chalabi 2, 3 Alberto Albanese 4 Peter Andersen 5 Maria del Mar Amador 6 Geir Bråthen Veronique Brunaud-Danel 7 Lev Brylev 8 William Camu 9 Mamede de Carvalho 10 Cristina Cereda Hakan Cetin 11 Delia Chaverri 12 Adriano Chiò 13 Philippe Corcia 14 Philippe Couratier 15, 16, 17 Fabiola de Marchi 18 Claude Desnuelle 19 Michael van Es 1 Jesús Esteban 20 Massimiliano Filosto 21 Alberto García Redondo 20 Julian Grosskreutz 22 Clemens Hanemann 23 Trygve Holmøy 24, 25 Helle Høyer 26 Caroline Ingre 27 Blaz Koritnik 28 Magdalena Kuzma-Kozakiewicz 29 Thomas Lambert 30 Peter Leigh 31 Christian Lunetta Jessica Mandrioli 32 Christopher Mcdermott 33 Thomas Meyer Jesus Mora 34 Susanne Petri 35 Mónica Povedano 36 Evy Reviers 37 Nilo Riva 38 Kit C.B. Roes 39 Miguel Á. Rubio 40 François Salachas 6 Stayko Sarafov 41 Gianni Sorarù 42 Zorica Stevic 43 Kirsten Svenstrup 44 Anette Torvin Møller 45 Martin Turner 46 Philip van Damme 47 Lucie A.G. van Leeuwen 1 Luis Varona 48 Juan Vázquez Costa 49 Markus Weber 50 Orla Hardiman 51 Leonard van den Berg 1, * 
* Auteur correspondant
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 (<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.
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https://hal-unilim.archives-ouvertes.fr/hal-03673852
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Soumis le : vendredi 20 mai 2022 - 11:29:38
Dernière modification le : lundi 5 septembre 2022 - 15:54:23

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Tessa Kliest, Ruben P.A. van Eijk, Ammar Al-Chalabi, Alberto Albanese, Peter Andersen, et al.. Clinical trials in pediatric ALS: a TRICALS feasibility study. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, Taylor & Francis, 2022, pp.1-8. ⟨10.1080/21678421.2021.2024856⟩. ⟨hal-03673852⟩

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