Nutritional Formulation for Angelman Syndrome

Completed · Not applicable · Has a placebo group

Conditions studied: Angelman Syndrome

In brief

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.

Key facts

Study ID

NCT03644693

Run by

University of Colorado, Denver

People needed

15

Starts

2018-11-01

Expected to finish

2020-01-15

Last updated by the study team

2020-08-20

Who can join

Age: 4 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female
• 4-11 years of age
• Diagnosed with Angelman syndrome with prior lab report indicating genetic diagnosis
• Currently on a LGIT, KD, or standard diet consistently for at least three months
• Motivated to maintain the LGIT or KD for at least the duration of the trial period, as applicable
• Written informed consent from patient and/or parent/caregiver
• Willing to drink provided formulations, or to eat them mixed with food
• Daytime toilet trained, or parents willing to use cotton balls in diapers to sample urine
• Willing to test urine daily
• Willing to comply with protocol examinations, including EEG, ERP, and venipuncture
• Ambulatory, willing to perform gait tracking

You may not qualify if…

• Being pregnant or planning pregnancy
• Requiring parenteral nutrition
• Major hepatic or renal dysfunction
• History of a diagnosis of diabetes
• Participation in other clinical intervention studies within 1 month prior to entry of this study
• Allergy to any of the study product ingredients
• Investigator concern around willingness/ability of patient or parent/caregiver to comply with protocol requirements
• Any contraindications for the use of the ketogenic or low carbohydrate diets
• Significantly underweight (Body Mass Index <18.5)
• Unwilling to drink provided formulations, or to consume formulation mixed with food
• Not ambulatory, or unwilling to perform gait tracking
• Not daytime toilet trained, or parents unwilling to use cotton balls in diapers to sample urine
• Unwilling to test urine daily

Where it is running

• Children's Hopsital Colorado — Aurora, Colorado, United States

Full record on ClinicalTrials.gov

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