Ketogenic Diet Program for Epilepsy

September 18, 2019 updated by: Ryan Lee, MD, Shriners Hospitals for Children
This study will assess the effectiveness of the ketogenic diet (high-fat, low-carbohydrate, and moderate protein) in treating epilepsy. Two study groups will be comprised of children with epilepsy (0-18 years of age) and whether or not they receive the ketogenic diet - epilepsy/ketogenic diet and epilepsy/non-ketogenic diet.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

According to an evidence-based guideline on the diagnosis and management of epilepsy from the National Institute for Clinical Excellence (2012), the ketogenic diet may be considered as an adjunctive treatment in children with drug-resistant epilepsy. Vast anecdotal and Class 1 studies have confirmed that the ketogenic diet helps most children with intractable seizures and cures many. Dietary therapies for epilepsy (e.g., classic ketogenic diet) have been shown to be highly effective. For example, Lee (P.R.) and Kossoff reported that approximately 50% of children with drug-resistant epilepsy had a greater than 50% reduction in seizures within days to months of treatment. In addition, use of the ketogenic diet as a treatment for epilepsy has been shown to reduce the escalating costs associated managing poorly controlled seizures (e.g., decrease in outpatient and emergency visits, inpatient hospitalization, neuroimaging, electro-encephalography, lab testing, and medication use).

If a child's seizures continue after two to three seizure medications have been tried, the ketogenic diet should be considered. However, many parents still medicate their children well beyond these guidelines and tolerate seizure frequency because they have no other alternatives. Given the physical and emotional toll that recurring seizures exact upon these children/families, the potential for improvement with the ketogenic diet is substantial. However, the ketogenic diet remains unavailable to most children.

Approximately 1% of Hawaii's children are projected to have epilepsy, but there is no established, ketogenic diet program for them to receive this dietary intervention, which can incorporate culturally distinct foods to improve palatability and compliance. Although the ketogenic diet has shown promise for broadening the scope of therapeutic options for children with epilepsy, it requires further study in an ethnically diverse population. At Shriners Hospitals for Children-Honolulu, the investigators have initiated a ketogenic and related dietary (e.g., modified Atkins diet) intervention program for children with epilepsy and started to assess its efficacy in treating epilepsy/seizures. This program also includes educational seminars and services to patients residing on the other Hawaii islands through outreach trips. The investigators have begun to enroll children with epilepsy into two groups based on whether or not they receive the ketogenic diet - epilepsy/ketogenic diet and epilepsy/non-ketogenic diet; total estimate of 15-30 participants over three years. Based upon initial findings, the investigators will implement a comprehensive, multidisciplinary ketogenic diet program that will potentially reach hundreds of children throughout Hawaii, the Pacific Basin, and elsewhere.

Specific Aims:

Aim 1. To assess the therapeutic efficacy of the ketogenic diet on epilepsy/seizures.

Hypothesis: Participants who have epilepsy/on the ketogenic diet will have significantly decreased number and severity of seizures than those that are not on the ketogenic diet, between baseline to three and six months after the dietary intervention is initiated.

Aim 2. In anticipation of lessened epilepsy/seizures, to determine the (a) change in number and dose of seizure medications used, (b) change in number of lab tests ordered for epilepsy management, (c) change in number of emergency room and hospital visits for seizures (or other neurodevelopmental problems), (d) change in number of neurologic procedures for epilepsy management (e.g. EEG, MRI, CT), and (e) participant/family satisfaction with the ketogenic diet.

Hypothesis: The number and/or dosage of medications, lab tests ordered, emergency room or hospital visits, and neurologic procedures for epilepsy management will decrease, and participant/family satisfaction will be high for participants who have epilepsy/on the ketogenic diet than those that are not on the ketogenic diet, between baseline to three and six months after the dietary intervention is initiated.

Aim 3. To compare differences and/or changes in (a) serum and urine ketone levels and (b) biochemical profiles as defined from blood and stool (gut or fecal microbiome) specimen samples.

Hypothesis: Participants who have epilepsy/on the ketogenic diet will have significantly higher serum/urine ketone levels and notably different biochemical profiles than those that are not on the ketogenic diet, between baseline to three and six months after the dietary intervention is initiated.

Children helped by the ketogenic diet are more likely to reach their highest level of functioning and become contributing adults. By providing the ketogenic diet as an intervention therapy for epilepsy in a safe and data-driven manner, the community-at-large will benefit and medical knowledge concerning dietary treatment for neurodevelopmental disorders will be advanced.

Study Type

Interventional

Enrollment (Actual)

30

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Hawaii
      • Honolulu, Hawaii, United States, 96826-1099
        • Shriners Hospitals for Children - Honolulu

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Ages 0-18 years.
  • Primary diagnosis of epilepsy.
  • Parent/legal guardian and child able to read or understand English, and able/willing to provide informed consent/assent.
  • Females of childbearing potential must have a negative pregnancy test result and agree to use a medically acceptable method of contraception throughout the entire study period and for 30 days after the last dose of study drug - childbearing potential is defined a girls who are > Tanner stage 2 and urine pregnancy tests are acceptable.

Exclusion Criteria:

  • Known cardiac disorder including arrhythmias or hypertension.
  • Carnitine deficiency (primary).
  • Carnitine palmitoyltransferase (CPT) I or II deficiency.
  • Carnitine translocase deficiency.
  • Beta-oxidation defects - medium-chain acyl dehydrogenase deficiency (MCAD), long-chain acyl dehydrogenase deficiency (LCAD), short-chain acyld dehydrogenase deficiency (SCAD), long-chain 3-hydroxyacyl-coenzyme A (CoA) deficiency, and medium-chain 3-hydroxyacyl-CoA deficiency.
  • Pyruvate carboxylase deficiency.
  • Porphyria.
  • Inability to maintain adequate nutrition.
  • Patient or caregiver non-compliance.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Epilepsy/Ketogenic Diet
Children (0-18 years of age) diagnosed with epilepsy will receive the ketogenic diet intervention.
Other: Ketogenic Diet
Dietary
No Intervention: Epilepsy/Non-Ketogenic Diet
Children (0-18 years of age) diagnosed with epilepsy will not receive the ketogenic diet intervention.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in the core symptoms of epilepsy (seizure frequency/severity)
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess the number of epileptic seizures through review/analysis of responses to the seizure log (self-report)
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in biochemical profiles due to the ketogenic diet
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess biochemical profile differences and changes through the analysis of blood and stool (gut microbiome) specimen samples
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Change from baseline in the number of medications used for epilepsy management
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess changes through the review/analysis of self-report and medical record data
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Change from baseline in the dosage of medications used for epilepsy management
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess changes through the review/analysis of self-report and medical record data
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Change from baseline in the number of lab tests ordered for epilepsy management
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess changes through the review/analysis of self-report and medical record data
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Change from baseline in the number of emergency room or hospital visits for epilepsy management
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess changes through the review/analysis of self-report and medical record data
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Change from baseline in subject/family satisfaction with the ketogenic diet
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess changes through the review/analysis of self-report and medical record data
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Change from baseline in ketone levels due to the ketogenic diet
Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)
Assess ketone level differences and changes through the analysis of serum and urine
Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shriners Hospitals for Children

Collaborators

University of Hawaii

Investigators

  • Principal Investigator: Ryan W Lee, MD, Shriners Hospitals for Children, Honolulu

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2015

Primary Completion (Actual)

January 19, 2018

Study Completion (Actual)

January 19, 2018

Study Registration Dates

First Submitted

July 9, 2015

First Submitted That Met QC Criteria

July 9, 2015

First Posted (Estimate)

July 14, 2015

Study Record Updates

Last Update Posted (Actual)

September 20, 2019

Last Update Submitted That Met QC Criteria

September 18, 2019

Last Verified

September 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HON1402

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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