Clinical trial NCT01248728: Omega-3 Fatty Acids For Treatment Of Young Children With Autism (OMG)
Official title: A Randomized, Placebo-Controlled Trial of Omega-3 Fatty Acids in the Treatment of Young Children With Autism
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: Autism Spectrum Disorder
Clinical Trial
Omega-3 Fatty Acids
Toddlers
Lead sponsor: Anagnostou, Evdokia, M.D.
Sponsor class: INDIV
Collaborators: name: Holland Bloorview Kids Rehabilitation Hospital
name: The Hospital for Sick Children
First posted: 2010-11-25
Enrollment: 38
Sex: ALL
Minimum age (years): 2
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a pilot, randomized, placebo-controlled trial of omega-3 fatty acids in autism. Autism, originally described by Kanner in 1943, is among the most severe of neurodevelopmental disorders. It is a Pervasive Developmental Disorder (PDD) affecting social and communicative functions and is also characterized by repetitive behaviors/restricted interests. It is also frequently accompanied by significant aggression, self-injury, irritability and hyperactivity, making care for these individuals an even greater challenge for families or institutional settings. Autism severely impacts the affected individual and family members, causing life-long functional impairment. In this protocol the investigators will use the terms "autism" and "autism spectrum disorder (ASD)" interchangeably to refer to Autistic disorder, Asperger Syndrome and PDD-Not Otherwise Specified (NOS).

Detailed description: Currently risperidone is the only medication approved the by Food and Drug Administration (FDA) for this disorder, and specifically for irritability associated with autism, although not all patients with autism respond to risperidone. No pharmacologic treatments have been approved for use in preschool children, although it is clear that early intervention is associated with improved outcomes. Behavioral and educational therapies play a significant role in the management of autistic symptoms. The history of alternative treatments in autism is notable for the exaggerated benefit of a variety of supplements, such as high dose vitamins (e.g. B6, magnesium), and secretin. The current widespread use of alternative/nutritional supplements to patients with autism without scientifically demonstrated efficacy, underscores the necessity for scientifically sound studies to be conducted. Complementary and alternative medical therapies (CAM) are commonly employed by families of autistic children. Recent surveys have estimated the prevalence of such use to be between 30% and 95% (1,2,3). Omega-3 fatty acids were reported to be used in 28.7% of patients (1). However, only two small case series and a very small randomized study have been reported in this population to date. The investigators propose to conduct a randomized controlled trial of omega-3 fatty acids in preschoolers with ASD.

Eligibility criteria: Inclusion Criteria:

1. Male or female outpatients 2-5 years of age.
2. Meet Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision (DSM-IV) criteria. DSM-IV criteria for an autism spectrum disorder, (Autistic disorder, Asperger syndrome or PDD-NOS) will be established by a clinician with expertise with individuals with ASD based on parent interview, Autism Diagnostic Observation Schedule (ADO) and Autism Diagnostic Interview (ADI-R)
3. If already receiving stable non pharmacologic educational, behavioral, dietary and or/ natural health product interventions during the preceding 3 months prior to Screening, will not electively initiate new or modify ongoing interventions for the duration of the study unless the child's condition is worsening or their turn comes up on the treatment waiting list.
4. Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
5. The parents must be able to speak and understand English sufficiently to allow for the completion of all study assessments.

Exclusion Criteria:

1. Patients born prior to 35 weeks gestational age.
2. Patients with any primary psychiatric diagnosis other than autism at Screening. We are aware the most primary psychiatric disorders are unlikely to be diagnosed in this age group
3. Patients with a medical history of neurological disease, including, but not limited to, epilepsy/seizure disorder (except simple febrile seizures), movement disorder, tuberous sclerosis, fragile X, and any other known genetic syndromes, or known abnormal MRI/structural lesion of the brain.
4. Patients with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease, or coagulation deficits.
5. Patients taking psychoactive medication(s) (e.g.,stimulants, antidepressants, antipsychotics, antiepileptics, anxiolytics, clonidine).
6. Patients that have been off pharmacotherapy for less than 6 weeks.
7. Patients who are participating in another clinical trial
8. Patients on anticoagulants
9. Patients who know that they will initiate or change nonpharmacologic interventions during the course of the study.
10. Patients unable to tolerate venipuncture procedures for blood sampling.
11. Patients taking Omega-3 supplements who have not discontinued treatment for six weeks prior to entering into the study.
12. Patients who have allergies to any of the ingredients in omega-3 (study product) or the placebo.

Primary outcomes:
measure: Change From Baseline in the Pervasive Developmental Disorder-Behavioral Inventory (PDDBI) - Autism Composite Score
timeFrame: Baseline and 24 Weeks
measure: Change From Baseline in the Behaviour Assessment System for Children (BASC-2) - Externalizing Problems Composite
timeFrame: Baseline and 24 Weeks
Secondary outcomes:
measure: Number of Participants Classified as Responders by the Clinical Global Impression - Improvement (CGI-I)
timeFrame: 24 weeks
measure: Change From Baseline in the Vineland Adaptive Behavioral Scales (VABS) - Adaptive Functioning Composite
timeFrame: Baseline and 24 Weeks
measure: Change From Baseline in the Preschool Language Scale (PLS-4) - Total Language
timeFrame: Baseline and 24 Weeks
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: Holland Bloorview Kids Rehabilitation Hospital
state: Ontario
Study officials:
affiliation: Holland Bloorview Kids Rehabilitation Hospital
name: Evdokia Anagnostou, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Mankad D, Dupuis A, Smile S, Roberts W, Brian J, Lui T, Genore L, Zaghloul D, Iaboni A, Marcon PM, Anagnostou E. A randomized, placebo controlled trial of omega-3 fatty acids in the treatment of young children with autism. Mol Autism. 2015 Mar 21;6:18. doi: 10.1186/s13229-015-0010-7. eCollection 2015.