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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

Title: 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
Authors: Cortese S.; McGinn K.; Hojlund M.; Apter A.; Arango C.; Baeza I.; Banaschewski T.; Buitelaar J.; Castro-Fornieles J.; Coghill D.; Cohen D.; Grunblatt E.; Hoekstra P. J.; James A.; Jeppesen P.; Nagy P.; Pagsberg A. K.; Parellada M.; Persico A. M.; Purper-Ouakil D.; Roessner V.; Santosh P.; Simonoff E.; Stevanovic D.; Stringaris A.; Vitiello B.; Walitza S.; Weizman A.; Wohlfarth T.; Wong I. C. K.; Zalsman G.; Zuddas A.; Moreno C.; Solmi M.; Correll C. U.
Contributors: Cortese, S.; Mcginn, K.; Hojlund, M.; Apter, A.; Arango, C.; Baeza, I.; Banaschewski, T.; Buitelaar, J.; Castro-Fornieles, J.; Coghill, D.; Cohen, D.; Grunblatt, E.; Hoekstra, P. J.; James, A.; Jeppesen, P.; Nagy, P.; Pagsberg, A. K.; Parellada, M.; Persico, A. M.; Purper-Ouakil, D.; Roessner, V.; Santosh, P.; Simonoff, E.; Stevanovic, D.; Stringaris, A.; Vitiello, B.; Walitza, S.; Weizman, A.; Wohlfarth, T.; Wong, I. C. K.; Zalsman, G.; Zuddas, A.; Moreno, C.; Solmi, M.; Correll, C. U.
Publication Year: 2023
Collection: Archivio della ricerca dell'Università di Modena e Reggio Emilia (Unimore: IRIS)
Subject Terms: Adolescent; Children; Dietary intervention; Medication; Probiotic; Psychopharmacology
Description: 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.
Document Type: article in journal/newspaper
Language: English
Relation: info:eu-repo/semantics/altIdentifier/pmid/37001575; info:eu-repo/semantics/altIdentifier/wos/WOS:001027082400001; volume:149; firstpage:105149_01; lastpage:105149_30; journal:NEUROSCIENCE AND BIOBEHAVIORAL REVIEWS; https://hdl.handle.net/11380/1315535
DOI: 10.1016/j.neubiorev.2023.105149
Availability: https://hdl.handle.net/11380/1315535; https://doi.org/10.1016/j.neubiorev.2023.105149
Rights: info:eu-repo/semantics/openAccess ; license:[IR] creative-commons ; license uri:http://creativecommons.org/licenses/by/4.0/
Accession Number: edsbas.40992D93
Database: BASE