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Clinical trials in pediatric ALS: a TRICALS feasibility study
Department of Neurology, UMC Utrecht Brain Centre, University Medical Centre Utrecht, Utrecht, Netherlands.
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.
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.
IRCCS Humanitas Research Hospital Rozzano, Milan, Italy.
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2022 (Engelska)Ingår i: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, ISSN 2167-8421, E-ISSN 2167-9223, Vol. 23, nr 7-8, s. 481-488Artikel i tidskrift (Refereegranskat) Published
Abstract [en]

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.

Ort, förlag, år, upplaga, sidor
Taylor & Francis Group, 2022. Vol. 23, nr 7-8, s. 481-488
Nyckelord [en]
clinical trial, clinical trials, ethics, Pediatric amyotrophic lateral sclerosis, pediatric investigation plan, therapy
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Neurologi
Identifikatorer
URN: urn:nbn:se:umu:diva-192954DOI: 10.1080/21678421.2021.2024856ISI: 000757188200001PubMedID: 35172656Scopus ID: 2-s2.0-85125309865OAI: oai:DiVA.org:umu-192954DiVA, id: diva2:1642652
Tillgänglig från: 2022-03-07 Skapad: 2022-03-07 Senast uppdaterad: 2022-12-30Bibliografiskt granskad

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