An Internet-Based Randomized Controlled Trial of Omega-3 Fatty Acids for Hyperactivity in Children with ASD

Background:   Complementary and alternative medical (CAM) therapies, such as omega-3 fatty acids, digestive enzymes, and high-dose vitamins are widely used to treat ASD despite little or no evidence of efficacy and safety. Traditional, clinic-based randomized controlled trials (RCTs) of therapies for ASD are expensive and extremely slow, often taking several years and many millions of dollars to complete. We sought to evaluate one of the more promising CAM therapies, omega-3 fatty acids, with a novel, fully internet-based clinical trial methodology to both evaluate the efficacy of omega-3 fatty acids and determine if internet-based RCTs (IB-RCTs) in ASD are feasible. Omega-3 fatty acids were selected because hyperactivity is a common problem among children with ASD and standard treatments (e.g., psychotropic medications) have unpredictable effects and more side effects in children with ASD. Two prior small pilot studies have found trends suggesting that omega-3 fatty acids may reduce hyperactivity in children with ASD.

Objectives: The goal of this study was to determine if the daily use of omega-3 fatty acids reduces hyperactivity compared to placebo in children with ASD and elevated baseline levels of hyperactivity. Secondary goals included assessments of change in social functioning, other ASD-related behaviors, and an evaluation of the performance of the IB-RCT.

Methods: This IB-RCT randomly assigned consented children ages 5-8 with ASD and elevated levels of hyperactivity to take 1.3 grams of omega-3 fatty acids daily vs. placebo over a 6-week period. The primary outcome measure was a comparison of the change in the hyperactivity subscale of the parent and teacher-administered Aberrant Behavior Checklist (ABC-H) between active and placebo groups. Secondary outcome measures included parent-completed Social Responsiveness Scales and Global Clinical Impression-Improvement scores.  All study procedures, including recruitment, informed consent, assessment of inclusion and exclusion criteria, and collection of baseline and outcome measures took place over the internet. The diagnosis of ASD was established by parent report and by a threshold score on the Social Communication Questionnaire, a method which has been validated in earlier studies. This study was conducted using IAN’s online tool, ORCA, which allows researchers to fully automate, track, and monitor   complex protocols.

Results: The study opened for enrollment on September 18, 2012.  As of October 9, 2012 (exactly three weeks into enrollment), 40 children from 22 U.S. states completed all enrollment procedures including engaging the child’s teacher. After being randomized into the control group or the treatment group (double-blind), these 40 subjects are currently involved in different stages the trial.  54 additional families have completed informed consent and are currently involved in the screening process.  The trial and data analysis is targeted for completion by the end of 2012.

Conclusions: The IB-RCT method is a faster and less expensive clinical trial design than a conventional clinical trial design for the evaluation of many safe interventions.  IB-RCTs can play a pivotal role in evaluating many commonly used, but unstudied interventions in ASD. Evidence from this study on the efficacy of omega-3 fatty acids will be available by end of 2012.