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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- dc.contributor.author Cieuta-Walti, Cécile
- dc.contributor.author Cuenca Royo, Aida Ma, 1981-
- dc.contributor.author Langohr, Klaus
- dc.contributor.author López-Vílchez, María Ángeles
- dc.contributor.author García García, Jordi
- dc.contributor.author Aldea-Perona, Ana
- dc.contributor.author Forcano, Laura
- dc.contributor.author Gomis González, Maria, 1988-
- dc.contributor.author Torre Fornell, Rafael de la
- dc.contributor.author PERSEUS Study Group
- dc.date.accessioned 2023-02-16T07:09:17Z
- dc.date.available 2023-02-16T07:09:17Z
- dc.date.issued 2022
- dc.description.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.
- dc.format.mimetype application/pdf
- dc.identifier.citation Cieuta-Walti C, Cuenca-Royo A, Langohr K, Rakic C, López-Vílchez MÁ, Lirio J, et al. 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). Genet Med. 2022 Oct; 24(10): 2004-13. DOI: 10.1016/j.gim.2022.06.011
- dc.identifier.doi http://dx.doi.org/10.1016/j.gim.2022.06.011
- dc.identifier.issn 1098-3600
- dc.identifier.uri http://hdl.handle.net/10230/55800
- dc.language.iso eng
- dc.publisher Elsevier
- dc.rights © 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/)
- dc.rights.accessRights info:eu-repo/semantics/openAccess
- dc.rights.uri http://creativecommons.org/licenses/by-nc-nd/4.0/
- dc.subject.keyword Children
- dc.subject.keyword Cognition
- dc.subject.keyword Down syndrome
- dc.subject.keyword EGCG
- dc.subject.keyword Safety
- dc.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)
- dc.type info:eu-repo/semantics/article
- dc.type.version info:eu-repo/semantics/publishedVersion