A phase 1, randomized double-blind, placebo controlled trial to evaluate safety and efficacy of epigallocatechin-3-gallate and cognitive training in adults with Fragile X syndrome
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- dc.contributor.author Torre Fornell, Rafael de la
- dc.contributor.author De Sola Llopis, Susana
- dc.contributor.author Xicota Vila, Laura, 1987-
- dc.contributor.author Cuenca Royo, Aida Ma, 1981-
- dc.contributor.author Rodriguez, Joan
- dc.contributor.author Gomis González, Maria, 1988-
- dc.contributor.author Hernández, Gimena
- dc.contributor.author Del Hoyo Soriano, Laura
- dc.contributor.author Ozaita Mintegui, Andrés, 1969-
- dc.contributor.author Dierssen, Mara
- dc.contributor.author TESXF Study Group
- dc.contributor.author Principe, Alessandro
- dc.contributor.author Espadaler, Josep María
- dc.contributor.author Roca, Laia
- dc.contributor.author Del Hoyo Soriano, Laura
- dc.contributor.author Farré Albaladejo, Magí
- dc.contributor.author Fitó Colomer, Montserrat
- dc.contributor.author León Jorba, Alba
- dc.contributor.author Banea, Ovideo
- dc.contributor.author Maldonado, Rafael, 1961-
- dc.contributor.author Busquets Garcia, Arnau, 1985-
- dc.date.accessioned 2019-04-30T07:50:39Z
- dc.date.issued 2020
- dc.description.abstract Background & aims: Despite the wide spectrum of experimental compounds tested in clinical trials, there is still no proven pharmacological treatment available for Fragile-X syndrome (FXS), since several targeted clinical trials with high expectations of success have failed to demonstrate significant improvements. Here we tested epigallocatechin-3-gallate (EGCG) as a treatment option for ameliorating core cognitive and behavioral features in FXS. Methods: We conducted preclinical studies in Fmr1 knockout mice (Fmr1−/y) using novel object-recognition memory paradigm upon acute EGCG (10 mg/kg) administration. Furthermore we conducted a double-blind placebo-controlled phase I clinical trial (TESXF; NCT01855971). Twenty-seven subjects with FXS (18–55 years) were administered of EGCG (5–7 mg/kg/day) combined with cognitive training (CT) during 3 months with 3 months of follow-up after treatment discontinuation. Results: Preclinical studies showed an improvement in memory using the Novel Object Recognition paradigm. We found that FXS patients receiving EGCG + CT significantly improved cognition (visual episodic memory) and functional competence (ABAS II-Home Living skills) in everyday life compared to subjects receiving Placebo + CT. Conclusions: Phase 2 clinical trials in larger groups of subjects are necessary to establish the therapeutic potential of EGCG for the improvement of cognition and daily life competences in FXS.
- dc.description.sponsorship This work was supported by the Spanish Ministry of Economy and Competitiveness; ‘Centro de Excelencia Severo Ochoa 2013-2017’; the ‘Centres de Recerca de Catalunya’ Programme / Generalitat de Catalunya’; the Centro de Investigación Biomédica en Red Enfermedades Raras (CIBER) of Rare Diseases; DIUE de la Generalitat de Catalunya [Grups consolidats SGR 2014/1125]; Ministerio de Economía, Industria y Competitividad de España MINECO [SAF2013-49129-C2-1-R and SAF2016-79956-R].
- dc.format.mimetype application/pdf
- dc.identifier.citation de la Torre R, de Sola S, Farré M, Xicota L, Cuenca-Royo A, Rodriguez J et al. A phase 1, randomized double-blind, placebo controlled trial to evaluate safety and efficacy of epigallocatechin-3-gallate and cognitive training in adults with Fragile X syndrome. Clin Nutr. 2020;39(2):378-87. DOI 10.1016/j.clnu.2019.02.028
- dc.identifier.doi http://dx.doi.org/10.1016/j.clnu.2019.02.028
- dc.identifier.issn 0261-5614
- dc.identifier.uri http://hdl.handle.net/10230/37158
- dc.language.iso eng
- dc.publisher Elsevier
- dc.relation.ispartof Clinical Nutrition. 2020;39(2):378-87
- dc.relation.projectID info:eu-repo/grantAgreement/ES/1PE/SAF2013-49129-C2-1-R
- dc.relation.projectID info:eu-repo/grantAgreement/ES/1PE/SAF2016-79956-R
- dc.rights © Elsevier http://dx.doi.org/10.1016/j.clnu.2019.02.028
- dc.rights.accessRights info:eu-repo/semantics/openAccess
- dc.subject.keyword Fragile-X syndrome
- dc.subject.keyword Epigallocatechin gallate
- dc.subject.keyword Cognition
- dc.subject.keyword Functionality
- dc.title A phase 1, randomized double-blind, placebo controlled trial to evaluate safety and efficacy of epigallocatechin-3-gallate and cognitive training in adults with Fragile X syndrome
- dc.type info:eu-repo/semantics/article
- dc.type.version info:eu-repo/semantics/acceptedVersion