NCT03644693

Nutritional Formulation for Angelman Syndrome

Evaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman Syndrome

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: University of Colorado, Denver · Academic / Other
• Sex: All
• Age: 4 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

Low carbohydrate diets such as the ketogenic (KD) and low glycemic index (LGIT) diets have been shown to be effective in treating drug resistant seizures in children with Angelman syndrome (AS). The investigators hypothesize that consuming a fat based nutritional formulation with an exogenous ketone throughout the day will produce urinary ketosis in children consuming both low and high carbohydrate diets, depending upon dietary background. The nutritional formulation will provide fuel substrates that push metabolism away from carbohydrates and towards fat utilization. This research is being done to assess the safety and tolerability of a nutritional formulation for use in dietary interventions in AS.

Detailed description

An evaluation of the safety and tolerability of a nutritional formulation in Angelman syndrome in children ages 4-11 years. Study is conducted over 16 weeks: 2 week baseline period - Patient continues on current dietary regimen they were on before joining the study (standard, ketogenic, or LGIT). 4 week intervention period - Patient consumes either placebo or nutritional formulation for 4 weeks, maintaining their current diet. 4 week washout period - Patient continues on current dietary regimen without consuming any study formulations. 4 week intervention period - Patient consumes either placebo or nutritional formulation for 4 weeks (crossover), maintaining their current diet. 2 week washout period - Patient continues on current dietary regimen without consuming any study formulations. Follow-up phone call to be conducted by study personnel during this time. Data is captured by the investigator during baseline, intervention, and washout periods, and by the parent/caregiver daily throughout the protocol using apps loaded on a provided tablet.

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	Nutritional Formulation Containing Exogenous Ketones	A nutritional formulation providing beta-hydroxybutyrate, 2g of carbohydrate, 1g of protein, and 9g of fat, plus minerals, per 100kcal. Given orally as food or beverage three times daily.
DIETARY_SUPPLEMENT	Placebo Formulation	A placebo formulation providing similar amounts of carbohydrates, proteins and minerals as the nutritional formulation. Given orally as food or beverage three times daily.

Timeline

• Start date: 2018-11-01
• Primary completion: 2020-01-15
• Completion: 2020-01-15
• First posted: 2018-08-23
• Last updated: 2020-08-20

Locations

1 site across 1 country: United States