Nutritional Approaches for Angelman Syndrome: Clinical Trial Update
Dr. Donna Herber and Dr. Jennifer Duis from Disruptive Enterprises presented an update on their clinical trial investigating the use of ketone supplements for the treatment of Angelman Syndrome at the 2018 FAST Science Summit. The trial aims to determine if ketone-based medical foods can improve nutrition, shift metabolism, and provide the benefits of a ketogenic diet without the restrictiveness. The trial design includes a double-blind, placebo-controlled crossover study with individuals aged 4 to 11. Primary outcomes focus on safety and tolerability, while secondary outcomes include measures of ketosis, sleep, seizures, neural activity, and mobility. The trial is actively recruiting participants, and data will be shared with the Global Registry for access by the community.
In this talk, we will provide an update on the clinical trial conducted by Dr. Donna Herber and Dr. Jennifer Duis from Disruptive Enterprises. The trial focuses on the use of ketone supplements for the treatment of Angelman Syndrome. The authors express their gratitude to the community for their support and collaboration in making this trial possible.
Dietary intervention has been used for almost 100 years to control seizures and epilepsy, with the ketogenic diet being particularly successful. In the case of Angelman Syndrome, both the ketogenic diet and low glycemic index diets have shown positive results in treating seizures. Additionally, ketone-based foods have been found to improve motor coordination, learning and memory, and seizures in animal models.
The Clinical Trial
The objective of the clinical trial is to determine if ketone-based medical foods can improve the nutrition and metabolism of patients with Angelman Syndrome, without the need for strict dietary restrictions. The trial is a collaboration between FAST and Disruptive Enterprises, with funding from the community.
The trial design includes a double-blind, placebo-controlled crossover study involving individuals aged 4 to 11. The primary outcome measures focus on safety and tolerability, with particular attention to gastrointestinal symptoms. Secondary outcomes include ketosis induction and maintenance, sleep improvement, seizure reduction, and changes in neural activity and mobility.
Trial Design and Initial Outcomes
The trial incorporates home monitoring and continuous data collection using iPads and electronic diaries. Blood and urine ketone measurements, as well as EEG, ERP, and gait tracking, are used to assess the effects of the ketone-based medical food. The trial is currently recruiting participants, and the data collected will be shared with the Global Registry for wider access.
The clinical trial on the use of ketone supplements for Angelman Syndrome is underway, with the primary focus on safety and tolerability. The trial aims to provide valuable insights into the effects of ketone-based medical foods on nutrition and metabolism in individuals with Angelman Syndrome. The authors express their appreciation to the community for their support and emphasize the importance of collaboration in advancing research and treatment options for Angelman Syndrome.
For more information or to participate in the trial, please contact the authors or visit their website.
- Title: Nutritional Approaches for Angelman Syndrome: Clinical Trial Update
- Author(s): Donna Herber, Jessica Duis
- Author(s)’ affiliation: Disruptive Nutrition
- Publication date: 2019-01-10
- Collection: 2018 FAST Science Summit