DURHAM, N.C.--(BUSINESS WIRE)--Disruptive Nutrition, the keto-medical foods company, a subsidiary of Disruptive Enterprises, today announced the first patient enrolled in a clinical research study that it is conducting in collaboration with leading investigators at Monroe Carell Jr. Children’s Hospital at Vanderbilt and the University of South Florida (USF). The study was funded by a grant from the Foundation for Angelman Syndrome Therapeutics (FAST).
This randomized, double-blind placebo controlled crossover study will assess safety and tolerability of a nutritional formula containing exogeneous ketones for use in dietary interventions for Angelman syndrome. Angelman syndrome is a rare genetic disorder caused by disruption in the ubiquitin-protein ligase E3A (UBE3A) gene.
“Initiation of this nutritional intervention represents a significant milestone for patients with Angelman syndrome, their caregivers and physicians,” said Donna Herber, PhD, chief science officer, Disruptive Enterprises. “This study demonstrates our continued commitment to improving societal wellbeing and scientific advancement of ketogenic technologies for nutritional support of Angelman syndrome and other medical conditions that would potentially benefit from a ketogenic diet. We are grateful for the families that will participate in this study and for our study partners at FAST, Vanderbilt and USF that are working with us on the scientific advancement of readily accessible and lifestyle appropriate ketogenic technologies.”
“For more than 10 years, FAST has been committed to the care of patients and families with Angelman syndrome with the goal of bringing practical treatment into current medical practice as quickly as possible,” said Allyson Berent, chief science officer, FAST. “The dedication shown by Disruptive and our academic study partners at Vanderbilt and USF on this innovative nutritional intervention has the potential to be a paradigm shift in the management of Angelman syndrome.”
“There is a significant unmet need for targeted nutritional support of patients with Angelman syndrome that could serve as adjunct therapy for the management of refractory seizures,” said Jessica Duis, MD, MS, Assistant Professor of Pediatrics at Children’s Hospital at Vanderbilt. “We expect that this study will generate important insights about ketogenic diets and how they can benefit Angelman syndrome patients. We look forward to continuing our partnership with Disruptive Nutrition, FAST and other academic centers to educate and advance the utilization of exogenous ketones in dietary interventions for a variety of patient populations.”
“We are excited to support this innovative clinical research study on a nutritional formulation containing exogenous ketones that would potentially transform the management of Angelman syndrome,” said Ed Weeber Professor, Molecular Pharmacology and Physiology, College of Medicine at the University of South Florida. For more information about the study, please visit Clinicaltrials.gov (Identifier, NCT 03644693)
About the Study
The study design is a 16 week, randomized, double blind, placebo-controlled crossover. Children age 4-11 with genetically confirmed AS, and a variety of dietary backgrounds (standard, low glycemic index, ketogenic) are eligible to participate. The protocol consists of a two-week baseline period, followed by the first four-week intervention period, a four-week washout period, cross over to the second four-week intervention period, and ending with a two-week washout. During baseline and intervention periods, children are assessed in the clinic for neurodevelopment, medical, seizure and dietary history, blood metabolism, EEG, cognition, and mobility.
About Angelman syndrome
Angelman syndrome is a rare genetic disorder caused by disruption in the UBE3A gene. It is characterized by developmental delays, including speech impairment and movement disorders. Eighty percent of patients have seizures, and a large proportion of the population also have feeding and sleep problems. The majority of children ages 4-11 with Angelman syndrome receive medication for seizures in addition to physical, occupational, and speech therapies. Some children with treatment-resistant seizures may also have special dietary interventions, such as the ketogenic diet. A low-carb/high-fat diet is a natural way to transition the brain to alternative sources of energy, which may decrease the excitability of the brain, thus minimizing seizure activity. By participating in this clinical study, children with Angelman syndrome may help medical researchers learn more about this genetic disorder and possible future treatment options.
For more information about Angelman syndrome, please visit: www.cureangelman.org
About Disruptive Enterprises, LLC
Disruptive Enterprises, LLC strives to be the science-based leader in education and innovation for readily accessible ketogenic technologies that boost the nutritional status and overall health and wellness across the life journey of consumers and patients. Disruptive leverages its experienced leadership and strong intellectual property to improve societal wellbeing by driving education and scientific advancement for delivery of readily accessible and lifestyle-appropriate ketogenic technologies.
FAST (Foundation for Angelman Syndrome Therapeutics) is a Section 501(c)(3) nonprofit research organization singularly focused on funding research that holds the greatest promise of treating Angelman syndrome. FAST is the largest, non-governmental funder of Angelman-specific research. Paula Evans, the mother of a young girl with Angelman syndrome, founded FAST in 2008. The foundation is based in Downers Grove, Ill. For more information about FAST, please visit https://cureangelman.org/.