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

Rafael de la Torre, Susana de Sola, Magí Farré, Laura Xicota, Aida Cuenca-Royo, Joan Rodriguez, Alba León, Klaus Langohr, María Gomis-González, Gimena Hernandez, Susanna Esteba, Laura del Hoyo, Júdit Sánchez-Gutiérrez, Maria José Cortés, Andrés Ozaita, Josep María Espadaler, Ramón Novell, Rafael Martínez-Leal, Montserrat Milá, Mara DierssenAlessandro Principe, Gonzalo Sánchez, Júdit Sánchez-Gutiérrez, Laia Roca, Rafasel de la Torre, Montserrat Fitó, Ovideo Banea, Maria José Cortés, Montserrat Milà, Rafael Maldonado, Arnau Busquets-Garcia, Andrés Ozaita, María Gomis-González

Research output: Contribution to journalArticleResearch

4 Citations (Scopus)

Abstract

© 2019 Elsevier Ltd and European Society for Clinical Nutrition and Metabolism 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.
Original languageEnglish
JournalClinical Nutrition
DOIs
Publication statusPublished - 1 Jan 2019

Keywords

  • Cognition
  • Epigallocatechin gallate
  • Fragile-X syndrome
  • Functionality

Fingerprint

Dive into the research topics of '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'. Together they form a unique fingerprint.

Cite this