Angelman Syndrome Clinical Trial
Official title:
Evaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman Syndrome
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.
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.
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