Effect of a Wide Spectrum Nutritional Supplement on Mitochondrial Function in Children With Autism Spectrum Disorder

Rossignol Medical Center16 enrolled

Overview

The objective of this study is to evaluate the metabolic effects of a comprehensive wide-spectrum supplement for children with ASD to determine whether it physiologically targets mitochondrial pathways known to be abnormal in children with ASD.The intervention is a commonly used wide-spectrum nutritional supplement, which is theoretically designed to normalize mitochondrial function. The investigators aim to determine if the supplement does have the hypothesized effect on physiology in individuals with ASD. The investigator will enroll up to 50 children, aged 4 to 14 years of age with confirmed ASD and mitochondrial dysfunction, and participation will last 26 weeks.

Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder often with life-long consequences that affects young children during critical developmental periods. The Centers for Disease Control estimates that ASD affects as many as 17 per 1000 children (1 in 59) in the United States suggesting that the prevalence is higher than previous estimates.Despite the dramatic rise in the detected prevalence of ASD over the past two decades, there is no effective medical treatment for core ASD symptoms (social communication and repetitive behavior), the closely associated problem of language impairment, or the underlying pathophysiology of ASD. Currently, the only accepted treatment for core ASD symptoms is behavior therapy, which may entail intensive one-on-one treatment over several years. The primary aims of this study are to evaluate the effect of a wide-spectrum nutritional supplement on mitochondrial function in individuals with ASD. Participants entered into the trial will have abnormalities in mitochondrial function that are known to be associated with ASD (approximately 50+% of children with ASD) but are not diagnostic of mitochondrial disease. The investigators hypothesize that nutritional supplements designed for children with ASD have a physiological action of normalizing mitochondrial function and cellular physiology throughout the body. To test whether the targeted nutritional supplement is superior to placebo, the investigators will study 50 children, between the ages of 4 years to 14 years, with confirmed ASD and known abnormal variations in mitochondrial at baseline. Participants will be randomly assigned to receive active treatment or placebo for 12-weeks under double-blind conditions and at the end of the 12 weeks switch to the opposite condition after \*no wash out period. Mitochondrial function will be measured at baseline and after each treatment arm in order to determine if the supplement positively influences cellular biochemistry. The investigator will also evaluate the effectiveness of the supplement on core and associated ASD symptoms using several behaviors assessments. \*note: 2 week wash out period was eliminated from the design to prevent attrition before the study was started. The CT.gov record was not updated for this change in the protocol in a timely manner. Published paper reflects the no wash out period. CT record is being updated for clarity. .

Eligibility

Sex: ALLMin age: 2 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Weight ≥ 15 kg and ≤ 100kg; * DSM-5 diagnosis of Autism Spectrum Disorder as established by formal clinical assessment which includes a gold-standard tool such as the Autism Diagnostic Observational Schedule. * Current Clinical Global Impression Severity score ≥ 4 * Stable educational and therapy plan (one month) with no planned changes in the intensity of treatment for 12 weeks. * English is spoken in the home and at least one parent is able to read, write and speak English. * Stable medication (no changes in past 6 weeks and no planned changes for the study duration. * Electron Transport Chain Complex (I, II, III, IV) or Citrate Synthase Activity which is \>= 2.0 Standard Deviation Above or Below Average (outside the normal range) Exclusion Criteria: * Presence of serious behavioral problems (tantrums, aggression, self-injury) for which another treatment is warranted. * Current Clinical Global Impression Severity score \< 7 (Extremely Ill) * Significant medical condition by history or by physical examination or lab tests that would be incompatible with the treatment. * Children taking anticonvulsant medication for seizures or active epilepsy. * Diagnosis of Mitochondrial Disease

Outcomes

Primary Outcomes

Change in Mitochondrial Activity in Study Patients: Citrate Synthase

Mitochondrial activity and redox metabolism at baseline and after the placebo and supplement arms of the study, as determined through laboratory assessment. Normal Range 4.4 to 22 with Mean and SD of 12.1 and 5.1. It is better to be within the normal range than outside of the normal range regardless of whether the value is higher or lower.