Clinical Trial: Ketogenic Diet Therapy for Autism Spectrum Disorder

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Ketogenic Diet Therapy for Autism Spectrum Disorder

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.

Detailed Summary:

Recent studies have shown that the ketogenic diet (high-fat, low-carbohydrate, and moderate protein; induce a shift from the primary metabolism of glucose to ketones) or Modified-Atkins diet may be effective in treating autism. Research on the Black and Tan BRachyury (BTBR) T+tf/J mouse strain, characterized by an autism-like behavioral phenotype, has demonstrated the efficacy of a ketogenic diet in improving autism. Although, modified diets, such as the Feingold diet, low-sugar diet, or gluten-free diet, have shown behavioral improvements in patients with Attention Deficit Hyperactivity Disorder (ADHD), the ketogenic diet has not been studied in autism spectrum disorder (ASD) despite the high incidence of ADHD comorbidity. The findings from only one prospective, pilot study have been published, which reported significant behavioral improvement in all 18 autistic subjects after six months (assessed at intervals of four weeks on the diet and two weeks diet-free) on the ketogenic diet. Clearly, the ketogenic and other modified diets as promising treatments for ASD have been understudied. Additional clinical research is necessary to establish the ketogenic diet as a safe, effective treatment alternative for children with ASD.

This proposed research project will involve an ethnically diverse sample (varied genetic background and environmental exposure) to assess the efficacy of the ketogenic diet as a therapeutic intervention, and to understand its beneficial effects in children with ASD. The investigators anticipate that noteworthy findings will contribute to the sparse literature on ASD and effective dietary interventions and prompt future research collaborations with investigators from other medical centers and/or academic institutions. Funding for future research appears promising considering that ketogenic therapies are also a novel method for the treatment of a variety
Sponsor: Shriners Hospitals for Children

Current Primary Outcome: 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).


Original Primary Outcome: 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: Asperger Syndrome Diagnostic Scale (ASDS), Childhood Autism Rating Scale (CARS-2), Gilliam Autism Rating Scale (GARS-3), Social Responsiveness Scale - Second Edition (SRS-2), standardized intelligence tests (if available, administered by child's school), and Vanderbilt ADHD Diagnostic Teacher Rating Scale Forms (Vanderbilt).


Current Secondary Outcome:

  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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
  • 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


Original Secondary Outcome:

  • Change from baseline in the number and 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.


Information By: Shriners Hospitals for Children

Dates:
Date Received: June 16, 2015
Date Started: March 2015
Date Completion: February 2018
Last Updated: February 28, 2017
Last Verified: February 2017