Details

Autor(en) / Beteiligte

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

Titel

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

Ist Teil von

• Clinical nutrition (Edinburgh, Scotland), 2020-02, Vol.39 (2), p.378-387

Ort / Verlag

England: Elsevier Ltd

Erscheinungsjahr

2020

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• 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. 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. 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. 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. [Display omitted]