| 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) |
| Authors: |
Cieuta-Walti, Cécile; Cuenca-Royo, Aida; Langohr, Klaus; Rakic, Claire; López-Vílchez, Ma Ángeles; Lirio, Julián; González-Lamuño Leguina, Domingo; González, Teresa Bermejo; García, Jordi García; Roure, Maria Rimblas; Aldea-Perona, Ana; Forcano, Laura; Gomis-Gonzalez, Maria; Cés, Sebastià Videla; Lacaille, Florence; Ravel, Aimé; Mircher, Clotilde; Walti, Hervé; Janel, Nathalie; Dairou, Julien; Lévy, Marilyne; Durand, Sophie; Dierssen, Mara; Sacco, Silvia; Fornell, Rafael de la Torre |
| Contributors: |
Institut Jérôme Lejeune; Universitat Politècnica de Catalunya = Université polytechnique de Catalogne Barcelona (UPC); Hospital Infantil Universitario Niño Jesús (HIUNJ); Marqués de Valdecilla Research Institute - Instituto de Investigación Marqués de Valdecilla Santander (IDIVAL); Hospital Universitari Sant Joan de Reus = Sant Joan de Reus University Hospital; Universitat Pompeu Fabra Barcelona (UPF); Universitat de Barcelona (UB); Gastro-Entérologie, Hépatologie et Nutrition pédiatriques (CHU Necker - Enfants Malades AP-HP ); Hôpital Necker - Enfants Malades AP-HP; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP); Unité de Biologie Fonctionnelle et Adaptative (BFA (UMR_8251 / U1133)); Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité); Laboratoire de Chimie et de Biochimie Pharmacologiques et Toxicologiques (LCBPT - UMR 8601); Institut de Chimie - CNRS Chimie (INC-CNRS)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité); Service de cardiologie pédiatrique CHU Necker; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Necker - Enfants Malades AP-HP; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP); Barcelona Institute of Science and Technology (BIST); CIBER de Enfermedades Raras (CIBERER); CHU Trousseau APHP; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU) |
| Source: |
ISSN: 1098-3600. |
| Publisher Information: |
CCSD; Nature Publishing Group |
| Publication Year: |
2022 |
| Subject Terms: |
EGCG; Safety; Down Syndrome; Double-Blind Method; Dietary Supplements; Cognition; Children; Catechin; Male; Humans; Child; Female; [SDV]Life Sciences [q-bio] |
| Description: |
International audience ; 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. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/35951014; PUBMED: 35951014 |
| DOI: |
10.1016/j.gim.2022.06.011 |
| Availability: |
https://cnrs.hal.science/hal-04281643; https://cnrs.hal.science/hal-04281643v1/document; https://cnrs.hal.science/hal-04281643v1/file/PIIS1098360022008206.pdf; https://doi.org/10.1016/j.gim.2022.06.011 |
| Rights: |
info:eu-repo/semantics/OpenAccess |
| Accession Number: |
edsbas.1940B38E |
| Database: |
BASE |