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Ketogenic Diet Therapy for Autism Spectrum Disorder

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ClinicalTrials.gov Identifier: NCT02477904
Recruitment Status : Active, not recruiting
First Posted : June 23, 2015
Last Update Posted : January 30, 2018
Sponsor:
Collaborator:
University of Hawaii
Information provided by (Responsible Party):
Ryan Lee, MD, Shriners Hospitals for Children

Brief Summary:
This study will assess the effectiveness of the ketogenic diet (high-fat, low-carbohydrate, and moderate protein) in treating autism spectrum disorder (ASD). Three study groups will be comprised of children (2-21 years of age) based on whether or not they have ASD and receive the ketogenic diet - ASD/ketogenic diet, ASD/non-ketogenic diet, and non-ASD/non-ketogenic diet.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Other: Ketogenic Diet Not Applicable

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 119 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Ketogenic Diet Therapy for Autism Spectrum Disorder
Study Start Date : March 2015
Estimated Primary Completion Date : February 2018
Estimated Study Completion Date : February 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: ASD/KD
Children (2-21 years of age) diagnosed with autism spectrum disorder (ASD) will receive the ketogenic diet (KD) intervention.
Other: Ketogenic Diet
Dietary
Other Names:
  • Classic Ketogenic Diet
  • Shriner's Ketogenic Diet
  • Non-Ketogenic Diet

Active Comparator: ASD/non-KD
Children (2-21 years of age) diagnosed with autism spectrum disorder (ASD) will not receive the ketogenic diet (KD) intervention.
Other: Ketogenic Diet
Dietary
Other Names:
  • Classic Ketogenic Diet
  • Shriner's Ketogenic Diet
  • Non-Ketogenic Diet

Active Comparator: non-ASD/non-KD
Typically developing children (2-21 years of age) diagnosed as not having autism spectrum disorder (ASD) will not receive the ketogenic diet (KD) intervention.
Other: Ketogenic Diet
Dietary
Other Names:
  • Classic Ketogenic Diet
  • Shriner's Ketogenic Diet
  • Non-Ketogenic Diet




Primary Outcome Measures :
  1. Change from baseline in core symptoms of ASD [ Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet) ]
    Assess core autistic symptoms through review/analysis of responses to the following measurement instruments: Autism Diagnostic Observation Schedule - Second Edition (ADOS-2); Asperger Syndrome Diagnostic Scale (ASDS); Childhood Autism Rating Scale (CARS-2); Gilliam Autism Rating Scale (GARS-3); Social Responsiveness Scale - Second Edition (SRS-2); Diagnostic and Statistical Manual IV Text Revision (DSM-IV-TR) and DSM-V ASD criteria; standardized intelligence tests (if available, administered by child's school); and Vanderbilt ADHD Diagnostic Teacher Rating Scale Forms (Vanderbilt).


Secondary Outcome Measures :
  1. Change from baseline in the number of medications used for ASD 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.

  2. Change from baseline in the dosage of medications used for ASD 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.

  3. Change from baseline in the number of lab tests ordered for ASD 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.

  4. Change from baseline in the number of emergency room or hospital visits for ASD management [ Time Frame: Pre- and post-ketogenic diet intervention (at baseline, and after three and six months on the ketogenic diet) ]
    Assess changes through review/analysis of self-report and medical record data.

  5. 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 in subject/family satisfaction with the ketogenic diet through review/analysis of responses to a questionnaire.

  6. 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 serum and urine ketone levels

  7. 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


Other Outcome Measures:
  1. Possible effects of the ketogenic diet [ Time Frame: Post-ketogenic diet intervention ]
    Participants on the ketogenic diet will be followed clinically with standard of care until they are 21 years of age to examine the long-term benefits or risks of the KD.



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Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Ages 2-21 years.
  • Primary diagnosis of autism spectrum disorder.
  • 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.
  • BMI < 3rd%ile.
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02477904


Locations
United States, Hawaii
Shriners Hospitals for Children - Honolulu
Honolulu, Hawaii, United States, 96826-1099
Sponsors and Collaborators
Shriners Hospitals for Children
University of Hawaii
Investigators
Principal Investigator: Ryan W Lee, MD Shriners Hospitals for Children - Honolulu

Publications:

Responsible Party: Ryan Lee, MD, Director - Neurodevelopmental Clinic, Research, Shriners Hospitals for Children
ClinicalTrials.gov Identifier: NCT02477904     History of Changes
Other Study ID Numbers: HON1403
First Posted: June 23, 2015    Key Record Dates
Last Update Posted: January 30, 2018
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Ryan Lee, MD, Shriners Hospitals for Children:
Autism
Autism Spectrum Disorder
Ketogenic Diet
Biochemical Profiles

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders