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http://hdl.handle.net/2445/191220
Title: | Safety and preliminary efficacy on cognitive performance and adaptive functionality of epigallocatechin gallate (EGCG) in children with Down syndrome. A randomized phase Ib clinical trial (PERSEUS study) |
Author: | Cieuta Walti, Cécile Cuenca Royo, Aida Langohr, Klaus Rakic, Claire López Vílchez, Ma Ángeles Lirio, Julián González Lamuño Leguina, Domingo Bermejo González, Teresa García García, Jordi Roure, Maria Rimblas Aldea Perona, Ana Forcano, Laura Gomis Gonzalez, Maria Videla Cés, Sebastià Lacaille, Florence Ravel, Aimé Mircher, Clotilde Walti, Hervé Janel, Nathalie Dairou, Julien Lévy, Marilyne Durand, Sophie Dierssen, Mara Sacco, Silvia Torre Fornell, Rafael de la Guerrico, Ion Álvarez Goday Arnó, Albert Llop, Cristina Mateus, Julián Matilla, Iris Menoyo, Esther Hernández Ligero, Lorena Pérez, Marta Pizarro, Neus Principe, Alessandro Cortesi, Anna Gomez, Alba Paredes, Noelia Gomez, Cristina Graell, Montserrat López, Pilar Lozano, Rosario Medrano, Constancio Andrade Navarro, Maria Teresa Daza, Gema Garcia, Sandra Gayán, Ma Luisa Guerra Colorado, Cristina Luna, Beatriz Maya, Enriqueta Millan, Delia Reina, Macarena Amigo, María Teresa Fernández, José Luís García Martínez, María Pérez Poyato, María Del Socorro Pozueta, Ana Tato, Emma Viadero, María Teresa Akkaya, Magnolia Falquero, Ségolène Gambarini, Alicia Akkaya, Magnolia Falquero, Ségolène Gambarini, Alicia |
Keywords: | Children Cognition Down syndrome Síndrome de Down Infants Down syndrome Children |
Issue Date: | 1-Oct-2022 |
Publisher: | Elsevier BV |
Abstract: | Purpose: Although some caregivers are using epigallocatechin gallate (EGCG) off label in hopes of improving cognition in young adults with Down syndrome (DS), nothing is known about its safety, tolerability, and efficacy in the DS pediatric population. We aimed to evaluate safety and tolerability of a dietary supplement containing EGCG and if EGCG improves cognitive and functional performance. Methods: A total of 73 children with DS (aged 6-12 years) were randomized. Participants received 0.5% EGCG (10 mg/kg daily dose) or placebo for 6 months with 3 months follow up after treatment discontinuation. Results: In total, 72 children were treated and 66 completed the study. A total of 38 participants were included in the EGCG group and 35 in the placebo group. Of 72 treated participants, 62 (86%) had 229 treatment-emergent adverse events (AEs). Of 37 participants in the EGCG group, 13 (35%) had 18 drug-related treatment-emergent AEs and 12 of 35 (34%) from the placebo group had 22 events. In the EGCG group, neither severe AEs nor increase in the incidence of AEs related to safety biomarkers were observed. Cognition and functionality were not improved compared with placebo. Secondary efficacy outcomes in girls point to a need for future work. Conclusion: The use of EGCG is safe and well-tolerated in children with DS, but efficacy results do not support its use in this population. (C) 2022 The Authors. Published by Elsevier Inc. on behalf of American College of Medical Genetics and Genomics. |
Note: | Reproducció del document publicat a: https://doi.org/10.1016/j.gim.2022.06.011 |
It is part of: | Genetics in Medicine, 2022, vol. 24, issue. 10, p. 2004-2013 |
URI: | http://hdl.handle.net/2445/191220 |
Related resource: | https://doi.org/10.1016/j.gim.2022.06.011 |
ISSN: | 1530-0366 |
Appears in Collections: | Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL)) |
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