| dc.contributor.author | Herber, Donna L. | |
| dc.contributor.author | Weeber, Edwin J. | |
| dc.contributor.author | D'Agostino, Dominic P. | |
| dc.contributor.author | Duis, Jessica | |
| dc.date.accessioned | 2020-10-22T16:38:44Z | |
| dc.date.available | 2020-10-22T16:38:44Z | |
| dc.date.issued | 2020-01-09 | |
| dc.identifier.citation | Herber, D. L., Weeber, E. J., D'Agostino, D. P., & Duis, J. (2020). Evaluation of the safety and tolerability of a nutritional Formulation in patients with ANgelman Syndrome (FANS): study protocol for a randomized controlled trial. Trials, 21(1), 60. https://doi.org/10.1186/s13063-019-3996-x | en_US |
| dc.identifier.other | eISSN: 1745-6215 | |
| dc.identifier.uri | http://hdl.handle.net/1803/16243 | |
| dc.description.abstract | Background Ketogenic and low-glycemic-index diets are effective in treating drug-resistant seizures in children with Angelman syndrome. Cognition, mobility, sleep, and gastrointestinal health are intrinsically linked to seizure activity and overall quality of life. Ketogenic and low-glycemic diets restrict carbohydrate consumption and stabilize blood glucose levels. The ketogenic diet induces ketosis, a metabolic state where ketone bodies are preferentially used for fuel. The use of exogenous ketones in promoting ketosis in Angelman syndrome has not been previously studied. The study formulation evaluated herein contains the exogenous ketone beta-hydroxybutyrate to rapidly shift the body towards ketosis, resulting in enhanced metabolic efficiency. Methods/design This is a 16-week, randomized, double-blind, placebo-controlled, crossover study to assess the safety and tolerability of a nutritional formula containing exogenous ketones. It also examines the potential for exogenous ketones to improve the patient's nutritional status which can impact the physiologic, symptomatic, and health outcome liabilities of living with Angelman syndrome. Discussion This manuscript outlines the rationale for a study designed to be the first to provide data on nutritional approaches for patients with Angelman syndrome using exogenous ketones. | en_US |
| dc.description.sponsorship | This project is funded by the Foundation for Angelman Syndrome Therapeutics in collaboration with Disruptive Nutrition, LLC. The Foundation reviewed a grant application substantially equivalent to the protocol described herein, but did not have direct input into protocol design or writing the manuscript. DLH is employed by Disruptive Nutrition and was involved in the design of the study and writing the manuscript. Neither the Foundation nor Disruptive Enterprises has ultimate authority over study design, collection, management, analysis, and interpretation of data; writing of the report, or publication; this responsibility rests with the trial sponsor, Vanderbilt University, and principal investigator, Dr. Duis. | en_US |
| dc.language.iso | en_US | en_US |
| dc.publisher | Trials | en_US |
| dc.rights | Copyright © The Author(s). 2020
Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated. | |
| dc.source.uri | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6953273/ | |
| dc.subject | Ketogenic diet | en_US |
| dc.subject | Ketosis | en_US |
| dc.subject | Angelman syndrome | en_US |
| dc.subject | Placebo-controlled trial | en_US |
| dc.subject | Children | en_US |
| dc.subject | Exogenous ketone | en_US |
| dc.subject | Ketone ester | en_US |
| dc.subject | Ketone supplement | en_US |
| dc.title | Evaluation of the safety and tolerability of a nutritional Formulation in patients with ANgelman Syndrome (FANS): study protocol for a randomized controlled trial | en_US |
| dc.type | Article | en_US |
| dc.identifier.doi | 10.1186/s13063-019-3996-x | |
