The future of child and adolescent clinical psychopharmacology: A systematic review of phase 2, 3, or 4 randomized controlled trials of pharmacologic agents without regulatory approval or for unapproved indications

Samuele Cortese*, Katherine McGinn, Mikkel Højlund, Alan Apter, Celso Arango, Immaculada Baeza, Tobias Banaschewski, Jan Buitelaar, Josefina Castro-Fornieles, David Coghill, David Cohen, Edna Grünblatt, Pieter J Hoekstra, Anthony James, Pia Jeppesen, Péter Nagy, Anne Katrine Pagsberg, Mara Parellada, Antonio M Persico, Diane Purper-OuakilVeit Roessner, Paramala Santosh, Emily Simonoff, Dejan Stevanovic, Argyris Stringaris, Benedetto Vitiello, Susanne Walitza, Abraham Weizman, Tamar Wohlfarth, Ian C K Wong, Gil Zalsman, Alessandro Zuddas, Carmen Moreno, Marco Solmi, Christoph U Correll

*Corresponding author af dette arbejde

    Publikation: Bidrag til tidsskriftReviewForskningpeer review

    Abstract

    We aimed to identify promising novel medications for child and adolescent mental health problems. We systematically searched https://clinicaltrials.gov/ and https://www.clinicaltrialsregister.eu/ (from 01/01/2010-08/23/2022) for phase 2 or 3 randomized controlled trials (RCTs) of medications without regulatory approval in the US, Europe or Asia, including also RCTs of dietary interventions/probiotics. Additionally, we searched phase 4 RCTs of agents targeting unlicensed indications for children/adolescents with mental health disorders. We retrieved 234 ongoing or completed RCTs, including 26 (11%) with positive findings on ≥ 1 primary outcome, 43 (18%) with negative/unavailable results on every primary outcome, and 165 (70%) without publicly available statistical results. The only two compounds with evidence of significant effects that were replicated in ≥ 1 additional RCT without any negative RCTs were dasotraline for attention-deficit/hyperactivity disorder, and carbetocin for hyperphagia in Prader-Willi syndrome. Among other strategies, targeting specific symptom dimensions in samples stratified based on clinical characteristics or established biomarkers may increase chances of success in future development programmes.

    OriginalsprogEngelsk
    Artikelnummer105149
    TidsskriftNeuroscience and Biobehavioral Reviews
    Vol/bind149
    DOI
    StatusUdgivet - jun. 2023

    Bibliografisk note

    Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.

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