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)

Cieuta-Walti, Cécile; Cuenca Royo, Aida Ma, 1981-; Langohr, Klaus; López-Vílchez, María Ángeles; García García, Jordi; Aldea-Perona, Ana; Forcano, Laura; Gomis González, Maria, 1988-; Torre Fornell, Rafael de la; PERSEUS Study Group

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)

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...

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Autores:	Cieuta-Walti, Cécile, Cuenca Royo, Aida Ma, 1981-, Langohr, Klaus, López-Vílchez, María Ángeles, García García, Jordi, Aldea-Perona, Ana, Forcano, Laura, Gomis González, Maria, 1988-, Torre Fornell, Rafael de la, PERSEUS Study Group
Tipo de recurso:	artículo
Estado:	Versión publicada
Fecha de publicación:	2022
País:	España
Institución:	Universitat Pompeu Fabra
Repositorio:	Repositorio Digital de la UPF
OAI Identifier:	oai:repositori.upf.edu:10230/55800
Acceso en línea:	http://hdl.handle.net/10230/55800 http://dx.doi.org/10.1016/j.gim.2022.06.011
Access Level:	acceso abierto
Palabra clave:	Children Cognition Down syndrome EGCG Safety

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spelling	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)Cieuta-Walti, CécileCuenca Royo, Aida Ma, 1981-Langohr, KlausLópez-Vílchez, María ÁngelesGarcía García, JordiAldea-Perona, AnaForcano, LauraGomis González, Maria, 1988-Torre Fornell, Rafael de laPERSEUS Study GroupChildrenCognitionDown syndromeEGCGSafetyPurpose: 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.Elsevier202320232022info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfapplication/pdfhttp://hdl.handle.net/10230/55800http://dx.doi.org/10.1016/j.gim.2022.06.011reponame:Repositorio Digital de la UPFinstname:Universitat Pompeu FabraInglés© 2022 The Authors. Published by Elsevier Inc. on behalf of American College of Medical Genetics and Genomics. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)http://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:repositori.upf.edu:10230/558002026-06-12T07:21:37Z
dc.title.none.fl_str_mv	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)
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)
spellingShingle	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) Cieuta-Walti, Cécile Children Cognition Down syndrome EGCG Safety
title_short	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)
title_full	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)
title_fullStr	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)
title_full_unstemmed	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)
title_sort	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)
dc.creator.none.fl_str_mv	Cieuta-Walti, Cécile Cuenca Royo, Aida Ma, 1981- Langohr, Klaus López-Vílchez, María Ángeles García García, Jordi Aldea-Perona, Ana Forcano, Laura Gomis González, Maria, 1988- Torre Fornell, Rafael de la PERSEUS Study Group
author	Cieuta-Walti, Cécile
author_facet	Cieuta-Walti, Cécile Cuenca Royo, Aida Ma, 1981- Langohr, Klaus López-Vílchez, María Ángeles García García, Jordi Aldea-Perona, Ana Forcano, Laura Gomis González, Maria, 1988- Torre Fornell, Rafael de la PERSEUS Study Group
author_role	author
author2	Cuenca Royo, Aida Ma, 1981- Langohr, Klaus López-Vílchez, María Ángeles García García, Jordi Aldea-Perona, Ana Forcano, Laura Gomis González, Maria, 1988- Torre Fornell, Rafael de la PERSEUS Study Group
author2_role	author author author author author author author author author
dc.subject.none.fl_str_mv	Children Cognition Down syndrome EGCG Safety
topic	Children Cognition Down syndrome EGCG Safety
description	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.
publishDate	2022
dc.date.none.fl_str_mv	2022 2023 2023
dc.type.none.fl_str_mv	info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion
format	article
status_str	publishedVersion
dc.identifier.none.fl_str_mv	http://hdl.handle.net/10230/55800 http://dx.doi.org/10.1016/j.gim.2022.06.011
url	http://hdl.handle.net/10230/55800 http://dx.doi.org/10.1016/j.gim.2022.06.011
dc.language.none.fl_str_mv	Inglés
language_invalid_str_mv	Inglés
dc.rights.none.fl_str_mv	http://creativecommons.org/licenses/by-nc-nd/4.0/ info:eu-repo/semantics/openAccess
rights_invalid_str_mv	http://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv	openAccess
dc.format.none.fl_str_mv	application/pdf application/pdf
dc.publisher.none.fl_str_mv	Elsevier
publisher.none.fl_str_mv	Elsevier
dc.source.none.fl_str_mv	reponame:Repositorio Digital de la UPF instname:Universitat Pompeu Fabra
instname_str	Universitat Pompeu Fabra
reponame_str	Repositorio Digital de la UPF
collection	Repositorio Digital de la UPF
repository.name.fl_str_mv
repository.mail.fl_str_mv
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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)

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